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SPECIAL ISSUE | PRODUCT GUIDE & EMERGING THERAPIES Volume 22 • Issue 1 • Speical Issue 2021 APPROVED PRODUCTS page 22 - EMERGING THERAPIES page 48 - COVID-19 VACCINES & EMERGING THERAPY ELIGIBILITY page 7
We’re counting down the days IN THIS ISSUE
ARTICLES
for HFA hugs to resume Assisting, educating, and advocating
for the bleeding disorders
COVID-19 Vaccines
& Emerging Therapies
community since 1994.
Volume 22 • Issue 1 • Special Issue 2021 Informed Consent:
EXECUTIVE LEADERSHIP
A Process, Not
Board Chair • Allie Ritchey Just A Form
President & CEO • Dr. Sharon
Meyers, CFRE
FDA's Gene
PRODUCTION TEAM
Editor • Kyle McKendall
and Cell Therapy
Design & Layout • Ashley Williams Framework
Proofreader • Melanie Padgett
Powers
Contributing Writers
Drug Recall
Michael Bishop, HFA Staff Process & Recent
Dr. David Clark Product Recalls
Dr. Lisa Hensley
Angela N. Johnson, PH.D
Lori Long, HFA Staff Drug Approval Process
Emily Roush-Bobolz, HFA Staff
Sonji Wilkes, HFA Staff
The information printed in Clinical Studies:
Dateline Federation is provided
for general purpose use. HFA does
How Do They
not give medical advice or engage Work?
in the practice of medicine and
recommends that you consult with your
physician or local treatment center
before beginning any form of treatment.
Requests for permission to translate
FEATURES
or reprint contents of Dateline
Federation should be directed to
info@hemophiliafed.org.
999 N. Capitol Street NE • Suite 301 Current
Washington, DC 20002
202.675.6984
Products
info@hemophiliafed.org for Treament
Until then, we’re here for ©2021 Hemophilia Federation of America.
Emerging
you and your family All rights reserved.
Connect with us on social media
for daily posts and updates about
Therapies
Undergoing
at www.hemophiliafed.org what’s happening at HFA. Clinical Trails
SPECIAL ISSUE 2021 3
DEAR FRIENDS—
One of our guiding principles since our founding has been to provide the tools and education
patients and their families need to make informed decisions about their bleeding disorder. This
annual special edition of Dateline Federation is a product of our focus on that principle. We hope
you find this tool helpful in your journey navigating treatment options and emerging therapies
and a conversation starter for you and your medical providers.
When the temperature rises above 86˚F
Esperoct has you covered
It was 17 years ago when I was choosing a treatment option for my newborn son with severe
a
®
hemophilia A. The overwhelming feeling remains vivid. It was a confusing and scary process,
despite having wonderful support from the staff at our local Hemophilia Treatment Center. I
The EHL product with the highest storage temperature for the longest time
was a mom, unsure of how to even spell hemophilia at that point, just wanting to make the right
call for my baby’s health. But, I didn’t know what questions to ask, what specifics to consider or
even what our options were. At that time, little did I know that the number of treatment options Be prepared with
for bleeding disorders would nearly double before my son even graduated high school and that
novel treatment options beyond a recombinant clotting factor would become readily accessible. Proven protection against bleeds High factor levels from one dose to the next
in adults and adolescents in adults and adolescentsc
As patients, we have the right to know our options. But it is our responsibility to educate and
• 1.2 overall bleeds per yearb • At or above 3% trough level for 100% of the timed
empower ourselves about our disorder. From our community’s history we know that patients and
EHL=extended half-life. • At or above 5% trough level for 90% of the time e
their families look to advocacy organizations such as HFA to provide the facts and information
a
For up to 3 months.
needed. HFA remains committed to being a trusted source of information for you. Furthermore, b
175 previously treated patients with severe hemophilia A received Esperoct ® 50 IU/kg every 4 days for 76 weeks based on median annualized bleed rates shown.
we are steadfast in our commitment to ensure a safe blood supply and transparency around c
Trough level goal is 1% for prophylaxis.
treatment options and their efficacy.
d
Data shown are from a study where 175 previously treated adolescents and adults received routine prophylaxis with Esperoct ® 50 IU/kg every 4 days for
76 weeks. Pre-dose factor activity (trough) levels were evaluated at follow-up visits. Mean trough levels for adolescents (12-
You may infuse ESPEROCT® at a hemophilia treatment Cap Color Indicator Nominal Strength
center, at your healthcare provider’s office or in your
home. You should be trained on how to do infusions Red 500 IU per vial
by your hemophilia treatment center or healthcare Green 1000 IU per vial
provider. Many people with hemophilia A learn to infuse
the medicine by themselves or with the help of a family Gray 1500 IU per vial
Brief Summary information about
ESPEROCT® [antihemophilic Factor member. Yellow 2000 IU per vial
(recombinant), glycopegylated-exei] Your healthcare provider will tell you how much Black 3000 IU per vial
This information is not comprehensive. ESPEROCT® to use based on your weight, the severity
COVID-19 VACCINES
• Talk to your healthcare provider or pharmacist of your hemophilia A, and where you are bleeding. Your Always check the actual dosage strength printed on
dose will be calculated in international units, IU. the label to make sure you are using the strength
• Visit www.novo-pi.com/esperoct.pdf to obtain prescribed by your healthcare provider.
FDA-approved product labeling Call your healthcare provider right away if your
DON’T STOP EMERGING
• Call 1-800-727-6500 bleeding does not stop after taking ESPEROCT®. How should I store ESPEROCT®?
If your bleeding is not adequately controlled, it could Prior to Reconstitution (mixing the dry powder in the
Patient Information be due to the development of Factor VIII inhibitors. This vial with the diluent):
ESPEROCT® should be checked by your healthcare provider. You Protect from light. Do not freeze ESPEROCT®.
[antihemophilic factor (recombinant),
glycopegylated-exei]
might need a higher dose of ESPEROCT® or even a
different product to control bleeding. Do not increase
Read the Patient Information and the Instructions the total dose of ESPEROCT to control your bleeding
®
ESPEROCT® can be stored in refrigeration at 36°F
to 46°F (2°C to 8°C) for up to 30 months until the
expiration date stated on the label. During the 30 month
THERAPY OPTIONS FOR THOSE
WITH BLEEDING DISORDERS
For Use that come with ESPEROCT before you ® without consulting your healthcare provider. shelf life, ESPEROCT® may be kept at room temperature
start taking this medicine and each time you get Use in children (not to exceed 86°F/30°C) for up to 12 months, or up
a refill. There may be new information. to 104°F (40°C) for no longer than 3 months.
ESPEROCT® can be used in children. Your healthcare
This Patient Information does not take the place of If you choose to store ESPEROCT® at room
provider will decide the dose of ESPEROCT® you will temperature:
talking with your healthcare provider about your medical receive.
condition or treatment. If you have questions about • Record the date when the product was removed BY HFA STAFF WITH SCIENTIFIC REVIEW
If you forget to use ESPEROCT®
ESPEROCT® after reading this information, ask your from the refrigerator. BY DR. DAVID CLARK AND DR. LISA HENSLEY
healthcare provider. If you forget a dose, infuse the missed dose when you • Do not return the product to the refrigerator.
What is the most important information I need to discover the mistake. Do not infuse a double dose to • Do not use after 12 months if stored up to 86°F
know about ESPEROCT®? make up for a forgotten dose. Proceed with the next (30°C) or after 3 months if stored up to 104°F Many people have questions about the COVID-19 against them. The first dose of the vaccine starts this
infusions as scheduled and continue as advised by your (40°C) or the expiration date listed on the vial,
Do not attempt to do an infusion yourself unless healthcare provider. whichever is earlier. vaccines. This article will answer one primary question: process and provides some protection. The second
you have been taught how by your healthcare Do not use this medicine after the expiration date which
provider or hemophilia treatment center. If you stop using ESPEROCT® Can getting vaccinated for COVID-19 cut off dose raises and refines that protection to about
is on the outer carton and the vial. The expiration date
You must carefully follow your healthcare provider’s Do not stop using ESPEROCT® without consulting your refers to the last day of that month.
instructions regarding the dose and schedule for healthcare provider. treatment options for emerging bleeding disorder 95% and helps the effects of the vaccine last longer.
After Reconstitution:
infusing ESPEROCT® so that your treatment will work If you have any further questions on the use of this treatments and therapies? Of the three vaccines The refinement is like the second time you play an
The reconstituted (the final product once the powder is
S:9.875"
B:11.5"
best for you. product, ask your healthcare provider.
T:11"
mixed with the diluent) ESPEROCT® should appear clear
What is ESPEROCT®? What if I take too much ESPEROCT®? and colorless without visible particles. currently available in the United States, the Pfizer and opponent in a sport: Even if they change some of their
ESPEROCT® is an injectable medicine used to replace Always take ESPEROCT® exactly as your healthcare The reconstituted ESPEROCT® should be used Moderna vaccines use messenger ribonucleic acid strategy, you are better able to handle them.
clotting Factor VIII that is missing in patients with provider has told you. You should check with your immediately.
hemophilia A. Hemophilia A is an inherited bleeding healthcare provider if you are not sure. If you infuse If you cannot use the reconstituted ESPEROCT® (mRNA) technology while the Johnson & Johnson
disorder in all age groups that prevents blood from more ESPEROCT® than recommended, tell your immediately, it must be used within 4 hours when The Johnson & Johnson vaccine works differently,
clotting normally. stored at or below 86ºF (30°C) or within 24 hours when
vaccine uses a viral vector.
healthcare provider as soon as possible.
ESPEROCT® is used to treat and prevent or reduce stored in a refrigerator at 36°F to 46°F (2°C to 8°C). using something called a “viral vector.” A viral vector
What are the possible side effects of
the number of bleeding episodes in people with
ESPEROCT®?
Store the reconstituted product in the vial. HOW THESE VACCINES WORK is created when scientists take a nonpathogenic
hemophilia A. Keep this medicine out of the sight and out of reach of
Your healthcare provider may give you ESPEROCT® Common Side Effects Include: children. The virus that causes COVID-19 is called SARS-CoV-2, virus (that doesn’t make humans sick) and alter it
when you have surgery. • rash or itching What else should I know about ESPEROCT® and
• swelling, pain, rash or redness at the location of
which is a coronavirus. Coronaviruses have protein to do something different. For the vaccine, Johnson
Who should not use ESPEROCT®? hemophilia A?
infusion Medicines are sometimes prescribed for purposes other spikes sticking out of them that allow the virus to & Johnson took an adenovirus and replaced part of
You should not use ESPEROCT® if you
Other Possible Side Effects: than those listed here. Do not use ESPEROCT® for a
• are allergic to Factor VIII or any of the other
You could have an allergic reaction to coagulation Factor condition for which it is not prescribed. Do not share attach to and enter cells. All three vaccines use that its genetic instructions with the genes to create the
ingredients of ESPEROCT®
VIII products. Call your healthcare provider right ESPEROCT® with other people, even if they have the spike protein to help our bodies develop an immune SARS-CoV-2 spike protein. When you get the shot, the
• if you are allergic to hamster proteins same symptoms that you have.
away or get emergency treatment right away if
If you are not sure, talk to your healthcare provider
before using this medicine.
you get any signs of an allergic reaction, such as: response to prevent illness. adenovirus enters your cells, giving them the genetic
hives, chest tightness, wheezing, dizziness, difficulty
Tell your healthcare provider if you are pregnant or Revised: 10/2019 instructions to make the spike protein. Once those
breathing, and/or swelling of the face.
nursing because ESPEROCT might not be right for you.
® ESPEROCT® is a trademark of In the case of the mRNA vaccines, when you get the
Your body can also make antibodies called “inhibitors” Novo Nordisk Health Care AG. spike proteins start appearing on the surfaces of your
What should I tell my healthcare provider before I against ESPEROCT®, which may stop ESPEROCT® from shot, mRNA goes into cells and sends “instructions” to
For Patent Information, refer to: http://novonordisk-us.
use ESPEROCT®? working properly. Your healthcare provider may need to com/patients/products/product-patents.html
cells, your immune system recognizes them as foreign
You should tell your healthcare provider if you: test your blood for inhibitors from time to time. the cells to create the spike protein. By itself, the spike
More detailed information is available upon and builds an immune response against them. As with
• Have or have had any medical conditions. These are not all of the possible side effects from request. Available by prescription only. protein will not cause COVID-19 — it’s just one piece
• Take any medicines, including non-prescription ESPEROCT®. Ask your healthcare provider for more Manufactured by: the mRNA, the viral vector with the DNA in it only lasts
medicines and dietary supplements. information. You are encouraged to report side effects Novo Nordisk A/S of the virus. The mRNA lasts in your system for about
• Are nursing. to FDA at 1-800-FDA-1088. Novo Allé a short time. This is because the viral vector does not
• Are pregnant or planning to become pregnant. DK-2880 Bagsværd, Denmark 24 hours and then starts to degrade, but your cells will
Tell your healthcare provider about any side effect that replicate well, so it also degrades out of the body. The
• Have been told that you have inhibitors to Factor VIII. bothers you or that does not go away. For information about ESPEROCT® contact: continue to express the spike proteins for a few days.
How should I use ESPEROCT®? What are the ESPEROCT® dosage strengths?
Novo Nordisk Inc. Johnson & Johnson vaccine requires only one dose at
800 Scudders Mill Road Your immune system recognizes those spike proteins
Treatment with ESPEROCT® should be started by a ESPEROCT® comes in five different dosage strengths. Plainsboro, NJ 08536, USA this time.
healthcare provider who is experienced in the care of The actual number of international units (IU) of Factor 1-800-727-6500 as foreign and starts creating an immune response
patients with hemophilia A. VIII in the vial will be imprinted on the label and on the © 2019 Novo Nordisk
ESPEROCT® is given as an infusion into the vein. box. The five different strengths are as follows: US19ESP00168 December 2019
SPECIAL ISSUE 2021 7
COMPARED TO GENE THERAPY
While the process is similar to what you have heard about gene
therapy for hemophilia, it is significantly different in the sense that Getting an mRNA vaccine
it uses a completely different viral vector and therefore would not will change your DNA.
interfere with you having access to gene therapy in the future. Other Getting an mRNA vaccine does
parameters in your health profile might keep you from getting gene not create any permanent genetic
therapy for hemophilia, but it won’t be because you had the vaccine. changes. mRNA is a piece of genetic
code that goes in, gives instructions
The Johnson & Johnson vaccine uses an adenovirus, which is to your cells, and, in the case of the
The vaccines can give
altered to create the protein spike. Gene therapy being studied for vaccine mRNA, starts leaving your
you COVID-19.
hemophilia treatment uses an adeno-associated virus (AAV). Even system in about 24 hours. It’s generally
though they sound the same, these two types of viruses are different gone in about three days. In addition, Because the vaccines
the vaccine mRNA works on the outer only use the SARS-CoV-2
and are not related, and having a Johnson & Johnson adenovirus
part of muscle cells without going into spike protein, there is
vaccine will not cause you to create antibodies to an AAV.
the cells’ nuclei, which is where your no way to get COVID-19
genes/DNA are located. from a vaccine.
SO, LET'S
BUST SOME
Getting an mRNA vaccine will
MYTHS:
prevent you from being able to
receive future bleeding disorder
treatments that use mRNA.
There is no evidence that having
an mRNA vaccine will close off SHARE
YOUR
access to future bleeding disorder
treatments that use mRNA. The
mRNA from the vaccine goes in
and gives instructions to make the
SARS-CoV-2 spike protein and
then is degraded in about 24 hours.
Getting a viral vector vaccine
will prevent you from being
able to receive future bleeding
STORY
disorder gene therapy treatments.
There is no evidence that having
a gene therapy vaccine made Connect with our IXINITY Ambassadors and share your experiences.
from an adenovirus will close off
access to future gene therapy Get started today at myIXINITYstory.com
bleeding disorder treatments
that use AAV. Medexus Pharma, Inc., Chicago, IL 60606
IXINITY® [coagulation factor IX (recombinant)] and any and all Medexus Pharma, Inc.
brand, product, service and feature names, logos, and slogans are trademarks or
registered trademarks of Medexus Pharma, Inc. in the United States and/or other countries.
©2021 Medexus Pharma, Inc. All rights reserved. MP-IX-0370
8 DATELINE FEDERATION < www.hemophiliafed.org
INFORMED CONSENT:
A Process, Not Just a Form
BY HFA STAFF
Patients with bleeding disorders are no regulations, an Institutional Review
strangers to engaging in conversations Board is an administrative body that
with medical, research and pharmaceutical has been formally designated to review
staff. Emergency rooms, doctors’ offices, and monitor biomedical research
pharmaceutical company offices, conference involving human subjects. The IRB
rooms and exhibit halls have all become places has the authority to approve, require
to engage in medical discussions. Each of these modifications needed to secure
venues has different, but similar, codes of ethics approval, or disapprove research. Tips for Understanding
to follow when engaging in conversations This group review serves an
about patients’ health, and all require obtaining important role in the protection of
Clinical Trials & Research Studies
informed consent before conducting health the rights and welfare of human
care intervention or research through a study research subjects. (The local We cannot emphasize enough that participants, sometimes referred to as human
or trial. IRB that approves the study subjects in a clinical research setting, should ask questions about their participation in
can provide more information a clinical trial or research study prior to signing up. Here are questions you should ask
Consent can only be obtained from a mentally about a clinical trial or research study and the information you should glean from asking:
about consent as it relates to
competent adult or a legally authorized
children.) WHAT SHOULD I ASK? my consent?
representative of a mentally incompetent • (In case of pharmaceutical trials) Will I be given
• Is a written copy of the research procedures
adult. This legal status refers to the capacity RESEARCH AND CLINICAL available? the actual medication or a placebo?
of a person to act on their own behalf and TRIALS • What are the benefits and risks of this research • (If English is not your first language) Is the
their ability to understand the information Consent works differently project? information available in my preferred language?
in a hospital setting • How will I be informed if there are changes to
presented, to appreciate the consequences of WHAT HAVE I LEARNED?
this project?
acting or not acting on that information, and to versus a research/ • The purpose of the research.
• Am I waiving any of my rights by signing this
make a choice. clinical trial setting. consent form?
• The name, address and phone number of the
Principal Investigator.
In the research/ • May I ask a person I trust to read this
• How long I am expected to participate.
PARTICIPATION BY MINORS clinical trial setting, document?
• If any of the medications or procedures are
A parent or legal guardian provides consent the first step to • Will my name and address be kept confidential
experimental.
or will it be shared with others?
for a minor. However, children 12 and older who understanding is • The possible risks or discomforts.
• How will my information be stored to protect
are asked to participate in a research project discovering that a my privacy?
• If there are any alternative procedures or
courses of treatment.
must do so voluntarily and must verbally research study or • May I refuse to participate in this research
• If my information and medical records will be
assent to the research project. The explanation clinical trial exists. project?
kept confidential.
of the project and the language used must be • What if I change my mind after I agree to
Patients may learn • If I will be paid for my participation.
participate?
appropriate to the child. In some localities, even about the existence • The medical treatments available if I get injured.
• How do I withdraw my consent after I sign the
children as young as 7 years old are asked to • Who I can call if I have questions.
of a research project form?
• If I am required to participate in this study.
assent to participate in research studies. or clinical trial via mail, • If I withdraw my consent and stop participating,
• If there is a penalty if I refuse to participate.
what will happen with the information already
telephone, through a friend • If there is a penalty if I stop participating at any
FDA REGULATIONS collected from me?
or during an in-person visit • Are there negative consequences if I withdraw
time.
Under the U.S. Food and Drug Administration
with a doctor.
More than a signature: Informed consent is about your understanding
10 DATELINE FEDERATION < www.hemophiliafed.org and willingness to participate in a study, not about signing a form.
SPECIAL ISSUE 2021 11
A LOOK AT FDA’S GENE AND
released as a draft in 2013 and guidance in 2017 and updated in gene therapies. This framework
finalized in 2015. It discussed 2020 to help industry apply these contained several new guidance
CELL THERAPY FRAMEWORK
many recommendations for new regulations that together documents, including hemophilia
manufacturing, testing and are referred to as the FDA CGT. This brought the total
patient safety and follow- regenerative medicine number of guidance documents
and its Impact on New Hemophilia
up. framework. Both human in the framework to 27. In 2021,
gene editing and transfer, we can see how the evolution
In 2016, Congress passed
Treatments in 2021 and Beyond the 21st Century Cures Act
as well as genetically
modified cells that
of hemophilia gene
therapy has been affected by the
and cell
or Cures Act, which includes
lead to a sustained factor development of this framework.
BY ANGELA N. JOHNSON, PH.D., RAC, CPGP provisions designed to expedite
production in hemophilia, are
SENIOR DIRECTOR OF REGULATORY AFFAIRS AT SIGILON THERAPEUTICS IN CAMBRIDGE, and streamline development of EARLY 2020: FDA LAUNCHES
MASSACHUSETTS, AND REGULATORY STRATEGY LECTURER AT NORTHEASTERN UNIVERSITY IN BOSTON considered to be regenerative CELL AND GENE THERAPY
innovative new medicines. This
medicine therapies by FDA. GUIDANCE FRAMEWORK
More cell and gene therapy as genetically modified factor- of guidance documents as a law builds on FDA’s existing
On Jan. 28, 2020, FDA launched
products are being developed producing cells do not involve playbook of how to do it in most, responsibilities and established Guidance for developing
its landmark guidance framework
and entering clinical trials each changes in patient genetic but not all, cases. regenerative medicine therapies, CGT products specifically for
for CGT products. Updates
year. The U.S. Food and Drug material and may allow better including gene therapy, cell hemophilia was published by
included improvements to the
Guidance frameworks are groups therapy, products made from FDA in 2018. Then in 2020, it was
Administration plays a key role control of dosing as well as guidance for hemophilia and
of related guidance documents, tissues and combinations of updated when FDA launched its
in overseeing drug development, redosing. But many challenges
such as those relating to CGT. New these products. FDA published expanded framework for cell and CONTINUED ON PAGE 17
including providing guidance and uncertainties face researchers
guidance creation is an important
and receiving investigational new and drug developers. To help set
part of FDA’s ongoing mission to
drug applications or requests best practices across the industry, GUIDANCE, REGULATION AND LAWS THAT MAKE UP
expedite innovations that make
to start a new clinical trial FDA guidance frameworks play THE U.S. FDA FRAMEWORK FOR DEVELOPING NEW CELL
medical products more effective,
submitted by drug developers. an important role in safe and AND GENE THERAPY (CGT) PRODUCTS FOR HEMOPHILIA
safer and more affordable.
efficient development of CGT
In 2021, there are more than Guidance documents are written Federal Food, Drug, & Cosmetic Act (FD&C)
products. The key law giving FDA responsibility to oversee safety of drugs, A look at the
1,000 cell and gene therapy and published according to a medical devices, and other products like food and cosmetics.
U.S. US FDA Framework
clinical trials, including more WHAT ARE FDA GUIDANCE process called Good Guidance Laws for Developing New
than a dozen in hemophilia. FDA FRAMEWORKS? Practice, which describes how Public Health Service Act (PHS)
Law that gives FDA more pathways and manufacturing information for
made by Congress
Cell & Gene Therapy
announced it expects more than To help the companies FDA staff will bring together biologic drugs, or drugs made from living organisms or their parts (e.g.
Products for
200 new requests to start clinical developing new drugs, FDA expert and public feedback in
genetic materials like DNA)
Regulations Hemophilia
trials based on gene and cell publishes recommendations guidance recommendations. (CFR Title 21)
21st Century Cures Act (CURES) of 2016
technologies each year. By called guidance documents. Law that creates regenerative medicine advanced therapy (RMAT)
GROWING EXPECTATIONS FOR
are made by FDA based on laws
framework, to speed development of innovative cell, gene, and tissue
Unlike the laws passed
2025, FDA expects 10 to CGT IN HEMOPHILIA
products.
20 new CGT treatments by Congress or formal FDA Guidance
FDA’s first draft guidance Coronavirus Aid, Relief, & Economic
will be approved annually. regulations, guidance Security Act (CARES) of 2020 are made by FDA with help from the public
document dedicated to
documents contain FDA In response to COVID-19 outbreak, gives FDA greater responsibility to help industry and the public interpret regulations
early development of CGT for managing interruptions & disruptions in drug manufacturing.
Gene therapy treatments expectations and current
clinical trials — Guidance for 2015 2017 2018 2020 GCT Framework:
for hemophilia have shown scientific thinking not required
Industry: Considerations for Regenerative Medicine Framework: Updated CGT Therapy for
potential to eliminate the need by law. If we think of regulations Design of Early
the Design of Early-Phase Phase CGT Trials Human Cell & Tissue Products
CGT Therapy for Hemophilia Guidance
CGT in Rare Disease Guidance
2021
Hemophilia Guidance
for prophylactic factor infusions as what the law requires drug Guidance Same Surgery Exception
Long-term CGT Trial Follow-up CGT
Clinical Trials of Cellular and RMAT Expedited Programs
CGT Manufacturing Guidance Manufacture
and injections. Unlike gene developers to do, we can think Medical Device Use with Products During COVID
Gene Therapy Products — was CGT “Sameness” for Orphan Drugs
therapy, newer technologies such
Image Source: Adapted for Sigilon Therapeutics, Inc. with permission of the author. © 2021 Angela N. Johnson. All rights reserved.
12 DATELINE FEDERATION < www.hemophiliafed.org SPECIAL ISSUE 2021 13
Summary of Selected Safety Information
What is the most important information I should know about SEVENFACT?
The most serious possible side effect of SEVENFACT is abnormal clotting involving blockage of blood
vessels, which include stroke, blockage of the main blood vessel to the lung, and deep vein blood clots.
You should know the signs of abnormal clotting and seek medical help immediately if they occur.
Signs of clotting in places other than your site of bleeding can include new onset of swelling and pain in
limbs, new onset of chest pain, shortness of breath, loss of sensation or motor power, or altered consciousness
or speech.
What is SEVENFACT?
*
SEVENFACT is an injectable medicine used for the treatment and control of bleeding episodes occurring in
adults and adolescents 12 years of age and older with Hemophilia A or B with inhibitors.
Injecting medicines requires special training; do not attempt to self-infuse unless you have been taught how
by your healthcare provider.
Who should not use SEVENFACT (coagulation factor VIIa)?
You should not use SEVENFACT if you are allergic to rabbits, or if you have known allergies to SEVENFACT or
any of its components. Seek immediate medical help if you experience hives, itching, rash, difficulty breathing
with cough or wheezing, swelling around the mouth and throat, tightness of the chest, dizziness or fainting, or
low blood pressure after taking SEVENFACT.
For the treatment of bleeding episodes in people* with hemophilia A or B with inhibitors
Tell your healthcare provider prior to using SEVENFACT if you have begun treatment of a bleeding episode
with another bypassing agent.
I’M READY TO MOVE ON What should I tell my healthcare provider before I use SEVENFACT?
Tell your healthcare provider if you are pregnant, are nursing, or plan to become pregnant; if you have had
prior blood clots, heart disease or heart failure, abnormal heart rhythms, prior pulmonary clots, or heart
surgery; or if you have or have had any other medical conditions.
What are the possible side effects of SEVENFACT?
Get rapid, predictable, and reliable The most common adverse reactions for SEVENFACT are headache, dizziness, infusion-site discomfort,
infusion-site hematoma, and infusion-related reaction and fever.
bleed control with SEVENFACT 225
†
Seek immediate medical help if you have signs of a blood clot or an allergic reaction.
To report SUSPECTED ADVERSE REACTIONS or product complaints, contact HEMA Biologics
Rapid effect: 3 hour at 1-855-718-4362. You may also report SUSPECTED ADVERSE REACTIONS to the FDA at
1-800-FDA-1088 or www.fda.gov/medwatch.
At 3 hours, 84% of mild/moderate bleeding episodes were controlled
with a single dose Please see Patient Product Information on the next page.
Predictable response: 84% ‡
At 9 hours, 84% of mild/moderate bleeding episodes treated achieved
bleed control after a single dose
Reliable control: 99.5% NEW
At 24 hours, 99.5% of mild/moderate bleeding episodes were resolved
Convenient home use: 98%
98% of bleeding episodes were treated at home Coagulation Factor VIIa
†
225 mcg/kg initial dosing regimen in the clinical trial.
(Recombinant)-jncw
As seen in the clinical trial.
Make the move
‡
PATIENT PRODUCT INFORMATION CONTACT YOUR HEALTHCARE PROVIDER IF YOU: rare disorders first drafted in many of the issues facing digital health and telemedicine
SEVENFACT® (SEV-en-fact) coagulation factor VIIa (recombinant)-jncw • Miss a dose, or
For Intravenous Injection After Reconstitution Only
• Administer more than your prescribed dose, or 2018, including Guidance for developers of cell and gene in 2020, and from the additional
• Think your bleed is not controlled within the expected time frame discussed with
PLEASE READ PATIENT PRODUCT INFORMATION AND THE INSTRUCTIONS FOR
your healthcare provider. Industry: Human Gene Therapy treatments for hemophilia and standardization of CGT
USE THAT COME WITH SEVENFACT BEFORE YOU START TAKING THIS MEDICINE
WHAT SHOULD I AVOID WHILE USING SEVENFACT?
AND EACH TIME YOU GET A REFILL. THERE MAY BE NEW INFORMATION.
• Avoid activity that can create more bleeding once you have completed your
for Hemophilia and Guidance other rare bleeding disorders. manufacturing practices under
This Patient Product Information does not take the place of talking with your healthcare
SEVENFACT infusion
for Industry: Human Gene the updated FDA guidance
THE NEW LANDSCAPE FOR
provider about your medical condition and treatment. If you have questions about
SEVENFACT after reading this information, ask your healthcare provider. • Avoid mixing SEVENFACT with other medications
Therapy for Rare Diseases. HEMOPHILIA CGT framework.
• Avoid infusing SEVENFACT and other factor-containing therapies [such as activated
WHAT IS THE MOST IMPORTANT INFORMATION I SHOULD KNOW
prothrombin complex concentrate (aPCC) or other recombinant Factor VIIa products]
ABOUT SEVENFACT?
at the same time. This increases your risk of having a disabling blood clot.
The most serious possible side effect of SEVENFACT is abnormal blood clotting involving
WHAT ARE THE POSSIBLE SIDE EFFECTS OF SEVENFACT?
FDA also confirmed draft In 2021, more than a dozen
blockage of blood vessels, which include stroke, blockage of the main blood vessel to the The CGT community has taken
lung, and deep vein blood clots. The most common adverse reactions reported in clinical trials for SEVENFACT were
headache, dizziness, infusionsite discomfort, infusion-site hematoma, infusion-related
recommendations, Guidance for gene transfer, gene editing and
You should know the signs of abnormal clotting (thrombosis) described below and seek
reaction and fever. important early steps on the
medical help immediately if they occur.
A serious allergic reaction to SEVENFACT may occur. If you experience the severe symptoms
Industry: Long Term Follow- Up genetically modified cell therapy
New onset of swelling and pain in the limbs or abdomen, new onset of chest pain, shortness
of an allergic reaction after infusing SEVENFACT, seek immediate medical attention. Severe path to harmonization or
of breath, loss of sensation or motor power, and altered consciousness or speech can all be
symptoms occur when your immune system reacts very strongly to foreign proteins or drugs. After Administration of Human trials for hemophilia are
signs of clot formation in places other than your site of bleeding. Seek immediate medical
• Hives, itching, rash, difficulty breathing with cough or wheezing, swelling around the development of international
attention if you experience one or more of these symptoms.
mouth and throat, tightness of the chest, dizziness or fainting, and low blood pressure Gene Therapy Products, to ongoing, with AAV gene
SEVENFACT should be used as prescribed and directed by your healthcare provider.
are all symptoms of a severe allergic reaction (anaphylaxis). Call 911 should you recommendations applicable
WHAT IS SEVENFACT? experience one or more of these symptoms. follow patients for safety therapies the furthest
SEVENFACT is a recombinant human Factor VIIa protein for injection. SEVENFACT can allow These are not all the possible side effects of SEVENFACT. For more information, ask across the world. However,
adolescents and adults with Hemophilia A or B with inhibitors to create clotting at the site of your healthcare provider or pharmacist. for five to 15 years after along though lentivirus
bleeding without needing Coagulation Factor VIII or IX replacement. several hemophilia gene therapy
SEVENFACT, coagulation factor VIIa (recombinant)-jncw, is indicated for the treatment and
Call your healthcare provider for medical advice about side effects. You may report
side effects to FDA at 1- 800-FDA-1088.
participating in a CGT gene therapy and non-
control of bleeding episodes occurring in adults and adolescents 12 years of age and older trials have also experienced
with Hemophilia A or B with inhibitors. HOW SHOULD I STORE SEVENFACT? clinical trial. viral genetically modified
Injecting medications requires special training. Do not attempt to self-infuse unless you have
been taught how by your health care provider or hemophilia treatment center. Once trained,
• SEVENFACT should be stored in its product packaging to protect from light. setbacks related to making or
you will need additional infusion materials along with your SEVENFACT so that you can
• Prior to reconstitution, the SEVENFACT kit should be stored at room temperature
cell therapies clinical trials.
successfully treat your bleeding episodes at home. Be sure to collect all necessary infusion
but can be stored between 36°F to 86°F (2°C to 30°C). The framework also updated distributing CGT products during
materials before starting the reconstitution process. • After reconstitution, SEVENFACT should be stored at room temperature but can be
The outbreak of the respiratory
SEVENFACT comes in a sterile dry powdered dosage form that must be reconstituted with
stored between 36°F to 86°F (2°C to 30°C), for up to 4 hours. guidance for best practices for the COVID-19 outbreak and
sterile Water for Injection. • SEVENFACT should not be frozen.
disease COVID-19 caused by
It is not known if SEVENFACT is safe and effective in children under 12 years of age. GENERAL INFORMATION ABOUT THE SAFE AND EFFECTIVE USE OF SEVENFACT manufacturers to make and test delays to clinical trials while new
This leaflet summarizes the most important information about SEVENFACT. the novel coronavirus SARS-
WHO SHOULD NOT USE SEVENFACT?
Do not use SEVENFACT for a condition for which it was not prescribed. Do not give
new CGT products, Guidance scientific and safety information
You should not use SEVENFACT if you:
SEVENFACT to other people even if they have the same symptoms you have. It may harm CoV-2, and passage of the
• Are allergic to rabbits. them. for Industry: Chemistry, is being investigated and shared
• Have known allergies to SEVENFACT or any of its components. You can ask your pharmacist or healthcare provider for information about SEVENFACT that Coronavirus Aid, Relief, &
Tell your doctor prior to infusing SEVENFACT if you have begun treatment of a bleeding is written for health professionals. Manufacturing, and Control with FDA. Such communication
episode with another bypassing agent such as activated prothrombin complex concentrate For more information, go to www.SEVENFACT.com or call 855.718.HEMA (4362). Economic Security Act of 2020
(FEIBA®).
WHAT ARE THE INGREDIENTS IN SEVENFACT?
Information for Human Gene between product developers
WHAT SHOULD I TELL MY HEALTHCARE PROVIDER BEFORE I USE SEVENFACT? by Congress has shifted the
Tell your healthcare provider if you:
Active ingredient: coagulation factor VIIa (recombinant)-jncw
Therapy, Investigational New and regulatory agencies, such
INACTIVE INGREDIENTS: ARGININE HYDROCHOLORIDE, GLYCINE, ISOLEUCINE,
focus of FDA. In January 2021,
• Are pregnant, planning to become pregnant or nursing, as SEVENFACT has not been
studied in patients with Hemophilia A or B with inhibitors who are pregnant or nursing.
LYSINE HYDROCHLORIDE, POLYSORBATE 80, TRISODIUM CITRATE DIHYDRATE,
HYDROCHLORIC ACID, NITROGEN AND WATER FOR INJECTION.
Drug Applications. Finally, a new as FDA, is a very important part
FDA added recommendations
• Had prior blood clots, heart disease, heart failure, abnormal heart rhythms, prior
pulmonary clots or heart surgery.
FOR INFORMATION CONTACT:
draft guidance was published of developing safe and effective
HEMA Biologics, LLC
for CGT manufacturers to its
• Have or have had any other medical conditions. 4441 Springdale Road
Louisville, Kentucky 40241-1086
to help clarify that products treatments. The COVID-19
You and your doctor can then decide whether SEVENFACT is the right treatment for you, as
guidance framework, Guidance
well as the proper timing and doses you will need for SEVENFACT to control your bleeding
episodes at home.
Manufactured by:
Laboratoire Francais du Fractionnement et des Biotechnologies S.A. (LFB S.A.)
treating rare disease must be outbreak could also increase
Les Ulis, 91940 for Industry: Manufacturing
HOW SHOULD I USE SEVENFACT?
France unique (i.e., not “sameness” the time it takes for these
Treatment with SEVENFACT should be started by a healthcare provider who is experienced
Distributed by: Considerations for Licensed
in the care of patients with Hemophilia A or B with inhibitors.
HEMA Biologics such as minor variations of the treatments to become widely
SEVENFACT is given as an injection into your vein. Louisville, KY 40241 and Investigational Cellular
You may infuse SEVENFACT at a hemophilia treatment center, at your healthcare provider’s U.S. License No. 2061 same cell or genetic material) available. While current guidance
office, or in your home. You should be trained on how to infuse by your healthcare provider
All trademarks are the property of their respective owners. and Gene Therapy Products
or hemophilia treatment center. Many people with inhibitors learn to infuse by themselves or
“SEVENFACT” is a trademark of LFB S.A.
to apply for orphan drug status, framework has helped streamline
with the help of a family member.
During COVID-19 Public Health
Treating at first sign of a bleed is important for bleed management. Your healthcare provider PATENT Information: https://hemabiodup.wpengine.com/patents/
which gives certain incentives development of CGT, we expect
will tell you how much Approved 04/2020
Emergency.
SEVENFACT to use based on your weight and when to administer SEVENFACT. like tax credits, FDA fee waivers that FDA will continue to work
CGT FOR HEMOPHILIA IN
To administer SEVENFACT:
• Collect all materials needed for your prescribed dose and rights to market a new closely with researchers,
• Follow the Instructions For Use guide to reconstitute the prescribed number
of SEVENFACT vials SEVENFACT is a registered trademark of LFB S.A. drug exclusively (or without 2021 AND BEYOND product developers and
• Infuse following your healthcare provider’s instructions, using infusion materials
from your pharmacy
HEMA Biologics, LLC is the exclusive licensee and distributor
of SEVENFACT in the United States and its territories. competitors selling the same Despite many challenges patient groups to further
©2020 HEMA Biologics, LLC HBSEV-00015. All rights reserved.
drug) for seven years after for ongoing clinical trials evolve the CGT guidance
approval, Guidance for Industry: in 2020, CGT treatments framework to help ensure
Interpreting Sameness of Gene for hemophilia are much that the next generation of
Therapy Products Under the closer to reaching patients. hemophilia treatments are
Orphan Drug Regulations. This Many clinical trials have developed rapidly, efficiently
guidance framework clarified benefited from wide adoption of and safely.
SPECIAL ISSUE 2021 17
Class III: Includes a situation in which use of or recall classification. FDA then places the notice of the
exposure to the product is not likely to cause recall in the FDA Weekly Enforcement Report. Nearly
adverse health consequences. all recalls implemented in the U.S. are begun on a
voluntary basis by the anything we can go to get.
Market withdrawal: When a product has a minor
violation that would not be subject to FDA If a manufacturer has voluntarily initiated a recall, it
legal action a “market withdrawal” occurs. The is the manufacturer’s responsibility to promptly notify
product is removed by the firm from the market each of its direct accounts. If the recall extends beyond
or the firm corrects the violation. direct accounts, then the direct accounts should be
instructed by the recalling manufacturer to contact
Medical device safety alert: Released in
A Look at the
sub-accounts that may have received the product.
circumstances in which a medical device may
Once all the accounts have been informed about the
present an unreasonable risk of substantial harm.
recall, they must promptly follow the recall strategy
DRUG RECALL
These situations also are considered recalls in
that was previously put in place for that account.
certain cases.
FDA Requested Recall
PROCESS
Each FDA recall follows specific timelines and
In urgent situations, FDA may request a recall. The
procedures depending upon the circumstances. For
request is directed to the manufacturer that has
example, each recall is initiated with a written order
the primary responsibility for making or marketing
BY HFA STAFF that states the violation, the product, lot and serial
the product. Class 1 category recalls are most often
numbers to be recalled, and the timeline for the
requested recalls. It is important to note FDA considers
recall. Each recall is unique and requires its own recall
an FDA requested recall to be manufacturer initiated.
strategy developed by the Center Recall Unit. The CRU
will consider how far the recall should extend, whether The associate commissioner for regulatory affairs
the public needs to be warned and if so, in what approves all recall requests from FDA. A letter outlining
It is important to pay manufacturer to recall a
geographical area, and the appropriate assessment the need for a recall is sent to manufacturer.
attention when a product product or mandates recall
for recall effectiveness. A recall designated voluntary, After a recall has begun, the
is recalled, but with all the requirements. The FDA’s role is
requested and mandatory depends on recall is entered in the Recall
different sources of information and to oversee the manufacturer’s recall
who initiates the process. Enterprise System. The RES
the different types of recalls, it can be confusing. strategy, monitor the recall for effectiveness and
Based upon the gravity is a database used by FDA
Recalls, designed to protect the public’s health, are classify the recall.
of the situation, FDA will to submit, update, classify
used as a way to deliver information to consumers in
issue a public warning. and terminate recalls.
an expeditious manner. RECALL CLASSIFICATION
FDA Mandated Recalls
A recall is an action taken by a manufacturer to Class I: Includes a health hazard situation in RECALL METHODS FDA’s authority to issue
remove a product (food, drugs, medical devices and which there is reasonable probability that the
Voluntary Recall: a mandatory recall is very
cosmetics) from the market, initiated either by the use of the product will lead to serious, adverse
Initiation of a Recall limited. Subjects of mandatory recalls
manufacturer or by request from the U.S. Food and health consequences or death.
by a Manufacturer can include devices, biological products, human tissue
Drug Administration. In either case, the manufacturer
Consistent with its responsibility to protect the public intended for transplantation, infant formula, tobacco
removes or corrects a product that is in the market Class II: Includes a potential health hazard
health from products that are defective or potentially products and food. FDA also has discretion to order
and in violation of FDA rules and regulations. In both situation in which use of, or exposure to, a violative
harmful, a manufacturer may voluntarily initiate a recall. a mandatory recall if it finds that a human cell, tissue
cases, the FDA considers the recall to be manufacturer product may cause temporary or medically
If a recall is manufacturer-initiated, FDA reviews the or cellular or tissue-based product is a source of
initiated. reversible adverse health consequences or
information provided by the manufacturer, conducts a dangerous infection to humans or does not adequately
where the probability of serious adverse health
Alternatively, an FDA-mandated recall, also known health hazard evaluation, classifies the recall and then protect against communicable disease.
consequences is remote.
as a mandatory recall, occurs when FDA orders a advises the manufacturer in writing of the assigned
18 DATELINE FEDERATION < www.hemophiliafed.org SPECIAL ISSUE 2021 19Recent Recalls of Products Indicated for
Treatment of Bleeding Disorders
Product Manufacturer Date Issue Scope Action taken
Voluntary pharmacy-level recall. Manufacturer stated “patients can continue to
Manufacturing deviation that occurred 1 lot in United States; other lots use product they may have. Although the potential for safety risk to patients is
Mononine CSL Behring January 21, 2021
during the filling process. worldwide considered low, it cannot be fully excluded.” Note: Product was discounted in
Septemeber 2020; product was expected to be available through mid-2021.
Some carton/package labels may have
Tranexamic acid Voluntary recall to the hospital/clinic level. Product is exclusively used in inpatient
Mylan September 1, 2020 been mislabeled as an unrelated product, 4 lots
injection setting.
Amiodarone HCl Injection.
Ferring Pharmaceuticals Product testing revealed low volume and Voluntary recall (initiated as pharmacy-level recall; subsequently extended to
Stimate nasal
(Distributor: CSL July 21, 2020 therefore above-specification All product worldwide consumer-level). Manufacturer does not anticipate product becoming available
spray
Behring) concentration of active ingredient before second half of 2023.
Internal manufacturer audit revealed “one
step did not proceed as expected.”
Manufacturer informed FDA during its
regular on-site inspection and received
VONVENDI Takeda February 25, 2020 “feedback on how [they] could improve.” 2 lots, 3425 vials, U.S. only Voluntary pharmacy level recall.
Manufacturer subsequently recalled vials
from lots manufactured during this period,
despite finding no impact on product
safety or efficacy.
Printing misalignment on label could lead Lots of all fill sizes (600, 1200,
Humate-P CSL Behring October 15, 2019 No recall. Drug information alert issued by manufacturer.
to confusion on dosage/potency 2400 Ius)
Particulate matter outside specifications No recall (regulators agreed there was no change in product benefit/risk profile).
Found in 1 batch during routine
Hemlibra Genentech October 5, 2019 found in vials (product deviation, not Genentech notified U.S., European, Canadian and Japanese health authorities in
inspection
contamination) March 2019.
Injection needles of incorrect length
Genentech (through 124 families and 92 health care
included in shipment to patients who
Hemlibra contract specialty September 21, 2019 providers; shipments involved No recall; Genentech notified FDA via standard U.S. drug safety reporting.
receive product through the Genentech
pharmacy Medvantx) took place over ~2 months
Patient Foundation
Mislabeling resulted in distribution of
2 lots, 900+ vials, distributed
Kogenate FS Bayer July 19, 2019 wrong product, wrong dosage, Class 2 voluntary recall to end user.
over period of 6 months
post-expiration
20 DATELINE FEDERATION < www.hemophiliafed.org SPECIAL ISSUE 2021 2143 APPROVED PRODUCTS
BY THE NUMBERS:
PRODUCTS BY TYPE
DISTRIBUTION
OF PRODUCTS BY Recombinant
56% 28%
MANUFACTURER clotting factor
TAKEDA
CSL BEHRING
NOVO NORDISK
14%
CURRENT PRODUCTS PFIZER, INC.
BAYER
Plasma-derived
clotting factor
AVAILABLE FOR TREATMENT GRIFOLS, INC
OCTAPHARMA USA, INC.
Non-factor
product 2%
OF BLEEDING DISORDERS FERRING
PHARMACEUTICALS
Bi-specific
antibody
Whether you or your child has just been diagnosed, or you’ve lived with a bleeding SANOFI GENZYME
disorder for decades, knowledge of treatment options is a key component of AKORN NUMBER OF PRODUCTS
being able to advocate for yourself and essential to having informed conversations PHARMACEUTICALS BY INDICATION
with health care professionals. To help patients and caregivers with the process of BIO PRODUCTS
navigating available treatment options, we’ve compiled a comprehensive list of all
therapies currently available and approved by the Food and Drug Administration.
LABORATORY USA, INC.
DR. REDDY’S
23 9
LABORATORIES FACTOR VIII FACTOR IX
Information in this issue should not be interpreted as medical advice. We encourage
GENENTECH
frequent dialogue with experienced health care professionals regarding your health
and the therapies used to treat your bleeding disorder. HEMA BIOLOGICS 4 7
KEDRION BIOPHARMA INHIBITOR vWD
MEDEXUS
7 2
RARE OTHER
MOST RECENTLY RELEASED PRODUCTS
YEAR RELEASED PRODUCT INDICATION MANUFACTURER
2020 SEVENFACT Inhibitor HEMA Biologics
Desmopressin Dr. Reddy’s
2020 Acetate Injection USP Factor VIII; vWD
(desmopressin acetate)
Laboratories
2019 Novo Nordisk Factor VIII ESPEROCT
22 DATELINE FEDERATION < www.hemophiliafed.org 2018 Bayer Factor VIII JiviHOW TO NAVIGATE
OUR PRODUCT CHARTS
LIST OF APPROVED PRODUCTS The pages that follow contain a comprehensive and exhaustive list of products that are
approved by the Food and Drug Administration for treatment of a bleeding disorder. For
Detailed product information can be found on the following pages, organized by indication. ease of navigation, the charts are published in sections by indication (Factor VIII, Factor IX,
Inhibitor, VWD, Rare or Other), with each containing the following categories of information:
Advate Eloctate NovoSeven RT
Takeda • Factor VIII Sanofi Genzyme • Factor VIII Novo Nordisk • Factor VIII
Product
Adynovate ESPEROCT NUWIQ Name used to market and sell the therapy.
Takeda • Factor VIII Novo Nordisk • Factor VIII Octapharma USA, Inc. • Factor VIII
Afstyla FEIBA NF Obizur Manufacturer
CSL Behring • Factor VIII Takeda • Inhibitor Takeda • Rare Company that produces and sells the therapy.
Alphanate FIBRYGA Profilnine
Grifols, Inc • Factor VIII;vWD Octapharma USA, Inc. • Rare Grifols, Inc • Factor IX Product Type
Aphanine SD HEMLIBRA REBINYN Indicated method used to create product.
Grifols, Inc • Factor IX Genetech • Factor VIII;Inhibitor Novo Nordisk • Factor IX
Alprolix Hemofil M Recombinate
Specific Product Type
Sanofi Genzyme • Factor IX Takeda • Factor VIII Takeda • Factor VIII Detailed classification of product type, if applicable.
Amicar Humate-P RiaSTAP
(amniocaproic acid - CSL Behring • Factor VIII;vWD CSL Behring • Rare Half Life
oral solution and tablets) Amount of time a product stays intact in the bloodstream
Akorn Pharmaceuticals • Other Idelvion Rixibis until its efficacy is halved.
CSL Behring • Factor IX Takeda • Factor IX
BeneFIX
Pfizer, Inc. • Factor IX IXINITY SEVENFACT FDA Approved
Medexus • Factor IX HEMA Biologics • Inhibitor
Coagadex Year the product was approved for treatment by FDA.
Bio Products Laboratory USA, Inc. • Jivi Stimate
Rare Bayer • Factor VIII (Desmopressin Nasal Spray)
CSL Behring • Factor VIII;vWD
Indications
Corifact Koate Bleeding disorder type/factor deficiency the therapy is intended to treat.
CSL Behring • Rare Kedrion Biopharma • Factor VIII Tretten
Novo Nordisk • Rare
We’ve made every effort to ensure the accuracy of the information in this list by using information
Cyklokapron Kogenate FS
(tranexamic acid injection) Bayer • Factor VIII Vonvendi directly from manufacturers and publicly available information from websites, such as FDA.
Pfizer, Inc. • Factor VIII; Factor IX Takeda • vWD We do not encourage community members to use one product over another, and we strongly urge
Kovaltry
DDAVP (Desmopressin) Wilate you to discuss your treatment options with qualified medical professionals.
Bayer • Factor VIII
Ferring Pharmaceuticals • Factor Octapharma USA, Inc • Factor
VIII;vWD Lysteda VIII;vWD Content in this issue is current as of March 2021. Given the fast-paced environment that manufacturers
(tranexamic acid tablets) and governmental agencies work within, some information could have changed since going to print.
Desmopressin Acetate Ferring Pharmaceuticals • Other Xyntha
Please refer to manufacturers’ or the FDA’s websites for the most up-to-date information.
Injection USP Pfizer, Inc • Factor VIII
(desmopressin acetate) Novoeight
Dr. Reddy's Laboratories • Factor Novo Nordisk • Factor VIII Xyntha/Xyntha Solofuse
VIII;vWD Pfizer, Inc • Factor VIII F I N D YO U R S E C TI O N
FACTOR VIII • 26 FACTOR IX • 30 INHIBITOR • 32 vWD • 32 RARE • 34
24 DATELINE FEDERATION < www.hemophiliafed.orgYou can also read
