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Explore the truths of people living with hemophilia (PwH) and change the conversation

admin April 7, 2024

This is a paid public announcement from Sanofi and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the Sanofi website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; review opt-in language from any company website before sharing any identifying information.

PwH know how much work goes into managing the physical effects of their condition, but are there unseen impacts that PwH may not share broadly?

We were curious about how PwH really felt, so we brought 8 individuals together to share their hemophilia truths, and this is what we learned.

The many moving stories we heard brought a simple truth to light; hemophilia management can have a daily impact on mental health, family life, and the ability to explore other aspects of their identity. Kyle, one of the individuals we spoke to, shared the following about his experience growing up with hemophilia: “I was told, ‘No playing sports; you should be bubble wrapped.’” Have you ever felt limited by hemophilia, like Kyle? If so, you’re not alone.

Let’s discover some truths: what kind of unseen impacts are other PwH facing?
For many people living with hemophilia, the burden goes beyond the bleed. They may experience emotional, physical, and logistical challenges before, during, and even after bleeding episodes.

Emotional challenges
Many PwH have demanding treatment schedules that may disrupt their daily lives. Missing school, work, social and recreational opportunities, and more can have a negative impact on their mental health and lead to feelings of social isolation.

PwH may also experience negative impacts on their self-perception, social lives, and relationships.

Physical challenges
PwH live with daily concerns about their levels of bleed protection and are often aware of their vulnerability to breakthrough bleeds.

PwH may also periodically experience varying degrees of pain due to bleeding events and accumulated joint damage.

Logistical challenges
Management of hemophilia can come with logistical demands such as refrigeration requirements, the need for reconstitution, difficulties with scheduling treatment, and traveling to their treatment centers. These types of logistical considerations can make daily life more difficult for PwH.

Have you been sharing your whole truth with your healthcare provider (HCP)? Speaking your whole truth can help you and your HCP:
  • –    Better understand the daily unseen impact of hemophilia
  • –    Improve your hemophilia management and help you meet your individual goals
  • –    Educate PwH and caregivers about hemophilia and its various treatment approaches
Are there any of these challenges that resonate with you that you might not have shared openly? It’s time to consider your whole experience with hemophilia and change the conversation.

Are you ready to change the conversation?
Discover more truths and how you can address your whole experience at MyHemophiliaTruth.com

© 2024 Genzyme Corporation. All rights reserved. Sanofi is a registered trademark of Sanofi or an affiliate. All other trademarks are the property of their respective owners. MAT-US-2400561-v1.0-02/2024

Is it time to FACTOR UP your hemophilia A management?

admin March 24, 2024

This is a paid public announcement from Sanofi and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to a Sanofi website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

Living with hemophilia A often means learning to live with unknowns—painful bleeds that can happen without warning, not knowing when a bleed might interrupt your plans, or when juggling treatment needs interrupts juggling, well, life itself.

But recent treatments may be able to change some of that—like ALTUVIIIO [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]. In 2023, Sanofi received FDA approval for ALTUVIIIO, a first-in-class Factor VIII replacement therapy that provides proven bleed protection for longer with once-weekly prophylaxis.

Here are 4 reasons to consider ALTUVIIIO:

  1. HIGHER SUSTAINED FACTOR LEVELS

ALTUVIIIO is engineered to last longer. With just one weekly infusion, Factor VIII levels remain in the near-normal to normal range (over 40%) for most of the week and stay above 18%,* on average, in adults.

*Average trough levels were 18% for adults 18 years and older, 9% for adolescents aged 12 years to under 18 years, 10% for children aged 6 years to under 12 years, and 7% for children aged 1 year to under 6 years.

†This is information from a study in 13 previously treated adults with severe hemophilia A that had the goal of comparing how long ALTUVIIIO, Adynovate® [Antihemophilic Factor (Recombinant), PEGylated], and Advate® [Antihemophilic Factor (Recombinant)] stayed in the body after 1 dose. Half-life was 43 hours for ALTUVIIIO, 15 hours for Adynovate, and 11 hours for Advate.
Adynovate and Advate are registered trademarks of Baxalta Incorporated, a Takeda company.
EHL=extended half-life; SHL=standard half-life.

ALTUVIIIO can be used not only for weekly prophylaxis but also for on-demand bleed control and perioperative management. Regardless of how you use it, you can expect the same infusion process

  1. STUDIED—AND PROVEN—BLEED PROTECTION

 Before we dive into the data, it’s helpful to know how ALTUVIIIO was studied and to understand its safety profile.

For one year, the XTEND-1 study looked at treatment in 159 adults and adolescents. Participants were divided into 2 groups. Both groups used mean and median annualized bleed rates (ABRs) to evaluate the efficacy of ALTUVIIIO. Finding people’s mean ABR was the primary goal of the study.

Safety evaluated in 159 people taking ALTUVIIIO in the XTEND-1 study showed that:

  • 21% of people had headache (33 people)
  • 16% of people had joint pain (26 people)
  • 6% of people had back pain (9 people)

ALTUVIIIO was also studied in the XTEND-Kids trial, in 67 previously treated male children under the age of 12. These children all received at least 1 dose of ALTUVIIIO.

In 67 children taking ALTUVIIIO in the XTEND-Kids study at the time of the interim analysis:

  • 1% of children had headache (1 child)

In XTEND-1 and XTEND-Kids, people taking ALTUVIIIO had:

  • 0 inhibitors
  • 0 serious allergic reactions

Although no inhibitors were found, and no serious allergic reactions occurred in clinical studies, inhibitors and serious allergic reactions are possible with ALTUVIIIO.

Group 1

This group consisted of 133 people aged 12 years and older who switched to once-weekly ALTUVIIIO prophylaxis from prior prophylaxis. This group included 1 female participant. Efficacy of prophylaxis was evaluated in 128 of these patients.

The primary outcome showed a mean of <1 (0.7) bleeds per year (the median ABR was 0).‡ Here’s a look at how the study measured bleed rates:

  • Median ABR was the middle number of all ABRs, when ABRs were ordered from least to greatest
  • Mean ABR was the average number based on everyone’s ABRs

In the pediatric study, routine prophylaxis with ALTUVIIIO resulted in a mean ABR of 0.5 and a median ABR of 0.‡

It’s also worth noting that 78 of the people in Group 1 participated in a separate study to measure their ABRs on their prior prophylaxis. These 78 people went from 3 bleeds to less than 1 bleed a year (mean ABR 0.7). That’s a powerful reduction of 77% in yearly bleeds!‡

And, over the 52 weeks on ALTUVIIIO prophylaxis, 64% of people had 0 bleeds.‡

Group 2

People in this group (26 participants) switched from prior on-demand therapy to ALTUVIIIO on demand for 26 weeks, and then to ALTUVIIIO prophylaxis for another 26 weeks.

This group also saw striking results. On average, people who switched from ALTUVIIIO on demand to ALTUVIIIO prophylaxis went from 21 bleeds to less than 1 bleed a year (mean ABR 0.7). That’s a 97% mean reduction in yearly bleeds.‡

And over the 26 weeks on ALTUVIIIO prophylaxis, 77% of people had 0 bleeds.‡

Both groups showed significant improvement in bleed protection with ALTUVIIIO prophylaxis.

‡Data based on treated bleeds.

  1. CONFIDENCE THAT YOUR JOINTS ARE PROTECTED

The XTEND-1 study also examined target joint bleeds. When evaluating joint results at 52 weeks in the 128 people who participated in the XTEND-1 study, 72% of people had 0 joint bleeds‡ on prophylaxis after switching to ALTUVIIIO. 100% of target joints were resolved.

Target joints:

  • Are 3 or more spontaneous bleeds in a major joint within a period of 6 consecutive months
  • Were considered resolved if 2 or fewer bleeds occurred in the target joint within 12 months

‡Data based on treated bleeds.

  1. THE FEWEST WEEKLY INFUSIONS AMONG FACTOR VIII PROPHYLAXIS TREATMENTS

While most people with hemophilia grow accustomed to infusing, fewer infusions are generally preferred. In studies, ALTUVIIIO clearly outlasted other Factor VIII replacement therapies, meaning ALTUVIIIO takes longer to be reduced by half in the body and therefore lasts for a longer period.

So instead of needing up to 4 infusions a week with other treatments, patients on ALTUVIIIO infused only once per week.

ALTUVIIIO offers the fewest weekly infusions among Factor VIII prophylaxis treatments.

§This is information from a study in 13 previously treated adults with severe hemophilia A that had the goal of comparing how long ALTUVIIIO, Adynovate® [Antihemophilic Factor (Recombinant), PEGylated], and Advate®[Antihemophilic Factor (Recombinant)] stayed in the body after 1 dose. Half-life was 43 hours for ALTUVIIIO, 15 hours for Adynovate, and 11 hours for Advate.
||Doses and dosing intervals may be adjusted.
Adynovate and Advate are registered trademarks of Baxalta Incorporated, a Takeda company.

Hear from other people living with hemophilia A who have made the switch.

Now that you’ve learned about how ALTUVIIIO can keep factor levels higher for longer and protect you from bleeds, you may be considering a conversation with your doctor about switching up your treatment plan. Our Evaluation Guide can help. It offers a list of helpful questions to help you jump-start the conversation. You can also connect with your local Sanofi Community Relations and Education (CoRe) Manager, who can share additional resources and provide education.

INDICATION

ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is an injectable medicine that is used to control and reduce the number of bleeding episodes in people with hemophilia A (congenital Factor VIII deficiency).

Your healthcare provider may give you ALTUVIIIO when you have surgery.

IMPORTANT SAFETY INFORMATION

What is the most important information I need to know about ALTUVIIIO?

Do not attempt to give yourself an injection unless you have been taught how by your healthcare provider or hemophilia center. You must carefully follow your healthcare provider’s instructions regarding the dose and schedule for injecting ALTUVIIIO so that your treatment will work best for you.

Who should not use ALTUVIIIO?

You should not use ALTUVIIIO if you have had an allergic reaction to it in the past.

What should I tell my healthcare provider before using ALTUVIIIO?

Tell your healthcare provider if you have had any medical problems, take any medications, including prescription and non-prescription medicines, supplements, or herbal medicines, are breastfeeding, or are pregnant or planning to become pregnant.

What are the possible side effects of ALTUVIIIO?

You can have an allergic reaction to ALTUVIIIO. Call your healthcare provider or emergency department right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash, or hives.

Your body can also make antibodies called “inhibitors” against ALTUVIIIO. This can stop ALTUVIIIO from working properly. Your healthcare provider may give you blood tests to check for inhibitors.

The common side effects of ALTUVIIIO are headache, joint pain, and back pain.

These are not the only possible side effects of ALTUVIIIO. Tell your healthcare provider about any side effect that bothers you or does not go away.

Please see full Prescribing Information.

sanofi

©2024 Genzyme Corporation. All rights reserved.
ALTUVIIIO and Sanofi are registered trademarks of Sanofi or an affiliate.
MAT-US-2401281-v1.0-03/2024

HEMGENIX®, etranacogene dezaparvovec-drlb, shows long-term durability, safety, and greater bleed protection versus factor IX prophylaxis at 3 years post-treatment

admin February 25, 2024

This is a paid public announcement from CSL Behring and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the CSL Behring website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will

Ongoing studies offer years of durability data
The clinical trials for HEMGENIX—the first-ever gene therapy for hemophilia B approved by the FDA—are set up so that those who have received HEMGENIX are monitored on an ongoing basis. Data has been published annually and have supported the consistent safety and efficacy of HEMGENIX. Studies for HEMGENIX began in 2018 with a phase 2b trial of 3 people with 5 years of data collected. The HOPE-B phase 3 trial began in 2020 with 54 people to evaluate the safety and efficacy of treatment. The FDA approved the treatment in 2022. HEMGENIX has been studied for over 5 years, with data continuing to be collected.

Clinical Trial Durations

In December 2023, CSL Behring released the three-year results from the phase 3 trial for HEMGENIX. In the ongoing HOPE-B study, results at 3 years confirmed the durability and safety of treatment with HEMGENIX following a one-time infusion in people living with hemophilia B, and will be studied up to 5 years.

HEMGENIX—a groundbreaking option
According to Dr. Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan and principal investigator of the HOPE-B pivotal trial:

“The long-term follow-up data from the HOPE-B study reinforces that a one-time treatment with HEMGENIX can produce elevated and sustained factor IX levels and reduce the rate of annual bleeds for years in people living with hemophilia B. Most importantly, the data show that nearly all the Phase III trial participants three years post-treatment with HEMGENIX have remained free from the need for regular prophylactic infusions, which is groundbreaking for the hemophilia B community.”

Are you ready for bleed protection that lasts for years instead of weeks? Talk to your doctor about the first-ever treatment option that offers your body the ability to make its own factor IX, and whose durability and safety has been confirmed for years.

IMPORTANT SAFETY INFORMATION

What is HEMGENIX?
HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of
adults with hemophilia B who:

    • Currently use Factor IX prophylaxis therapy, or

    • Have current or historical life-threatening bleeding, or

    • Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is administered as a single intravenous infusion and can be administered only once.

What medical testing can I expect to be given before and after administration of HEMGENIX?
To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.

HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.

What were the most common side effects of HEMGENIX in clinical trials?
In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.

What should I watch for during infusion with HEMGENIX?
Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest lightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.

What should I avoid after receiving HEMGENIX?
Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.

Please see full prescribing information for HEMGENIX.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.

HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.
HEMGENIX® is a registered trademark of CSL Behring LLC.
©2024 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA
www.CSLBehring.com USA-HGX-0611-FEB24

The differences between gene therapies for hemophilia A and hemophilia B

admin November 27, 2023

This is a paid public announcement from CSL Behring and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the CSL Behring website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

Hemophilia is a genetic condition
Both hemophilia A and B are caused by mutations in the gene for blood clotting factor. Hemophilia A is caused by a mutation in the gene that creates factor VIII (FVIII) and hemophilia B is caused by a mutation in the gene that creates factor IX (FIX). Both the F8 and F9 genes are located on the X chromosome at different points.

Low factor levels lead to the inability of blood to clot, resulting in numerous physical and lifestyle burdens, including unexpected breakthrough bleeds and other chronic health problems. Over half the people with hemophilia A or B have factor levels less than 1% of normal.

Gene therapies for hemophilia A and B target different genes
Gene therapy is a long-term treatment option for people with hemophilia that offers extended bleed protection, which could eliminate the need for prophylaxis. Gene therapy uses an innovative approach that redefines treatment by either introducing a functioning gene into the body, or turning off or changing the gene that is causing the condition. Current gene therapies approved for hemophilia introduce a new, fully functioning gene into the body. The mutations causing hemophilia A and B have been characterized in thousands of people, and it is clear from the large number of mutations that the molecular basis of the condition is extremely diverse.

There are differences between gene therapies for hemophilia A and B
All gene therapy for hemophilia targets the liver. However, since there are differences in how the body produces FVIII and FIX, there are also fundamental differences in how gene therapy works in the liver.

For people with hemophilia B, gene therapy targets liver cells, known as hepatocytes, where factor IX proteins are naturally made. By delivering a functional F9 gene straight to the liver, it enables a person to start creating their own factor IX proteins that are missing or not working and causing the disorder.

In hemophilia A, a functional F8 gene is delivered to the liver, allowing it to start creating the missing or nonworking factor VIII proteins that cause the disorder. However, the way gene therapy for hemophilia A works is slightly different, since FVIII is produced by different liver cells and tissues than those that produce FIX. The F8 gene is larger and structurally complex, which creates additional challenges.

How gene therapy works
Working genes are usually delivered into the cells of the body by inserting them into an inactive viral shell, known as the vector.

Vectors being used in research are commonly made from adeno-associated viruses (AAVs). The AAV, naturally existing in the world at large, is deactivated, eliminating its ability to cause any illness while it performs its new task to deliver a therapy. In AAV-based gene therapy or gene transfer, a working gene is inserted into an AAV vector. An AAV vector protects and delivers the new gene to its destination through a one-time infusion. Current gene therapies for hemophilia A and B use different AAV vectors to deliver that new gene.

The size and simplicity of the F9 gene made it a promising target for gene therapy
Hemophilia B has long been a promising target for gene therapy because it is caused by a single gene mutation, which is both small in size and structurally simpler in comparison to hemophilia A.

In 2022, HEMGENIX®, etranacogene dezaparvovec-drlb, was approved by the FDA as the first and only gene therapy for hemophilia B. A one-time dose of HEMGENIX has been shown to offer elevated factor IX levels for years, with 37% average factor IX activity sustained at 2 years. HEMGENIX also offers greater bleed protection than prophylaxis. In a clinical trial, annualized bleed rate (ABR) for all bleeds decreased by 54% from an average of 4.1 for patients on prophylaxis during the lead-in period to 1.9 in months 7–18 after treatment. And 94% of, or 51 out of 54, people remained entirely free of continuous routine factor IX prophylaxis.

Hemophilia A has been a more challenging target for gene therapy

Due to constraints with AAV vectors, hemophilia A proved to be a challenging target for gene therapy. However, that changed recently, when the first gene therapy for hemophilia A was approved by the FDA. Administered as a single dose, gene therapy for hemophilia A has been shown to increase blood levels of factor VIII and reduce the risk of uncontrolled bleeding vs prophylaxis.

With gene therapies being approved for both hemophilia A and B, the future treatment landscape has irrevocably changed for anyone managing the condition.

IMPORTANT SAFETY INFORMATION

What is HEMGENIX?

HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:

  • Currently use Factor IX prophylaxis therapy, or
  • Have current or historical life-threatening bleeding, or
  • Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is administered as a single intravenous infusion and can be administered only once.

What medical testing can I expect to be given before and after administration of HEMGENIX?

To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.

HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.

What were the most common side effects of HEMGENIX in clinical trials?

In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.

What should I watch for during infusion with HEMGENIX?

Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.

What should I avoid after receiving HEMGENIX?

Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.

Please see full prescribing information for HEMGENIX.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch , or call 1-800-FDA-1088.

You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.

HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.

HEMGENIX® is a registered trademark of CSL Behring LLC.

©2023 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA

www.CSLBehring.com   USA-HGX-0466-NOV23

Gene therapy for hemophilia B offers long-term bleed protection with a one-time infusion

admin September 24, 2023

This is a paid public announcement from CSL Behring and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the CSL Behring website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

The current standard of care for moderate to severe hemophilia B, factor IX prophylactic therapy, does offer bleed protection. However, it requires lifelong, routine infusions to maintain protective factor levels, and those who regularly infuse factor IX replacement products can still experience breakthrough bleeds, leading to joint damage, pain, and reduced quality of life. There is a need for a treatment option that offers consistent bleed protection with a one-time infusion that lasts years instead of weeks.

After years of scientific research and clinical studies, that option is here—HEMGENIX® (etranacogene dezaparvovec-drlb), the first and only gene therapy for hemophilia B.*

Hemophilia B is an appropriate target for treatment with gene therapy

Hemophilia B is caused by a mutation of a single gene—the F9 gene. Approaches using gene therapy to treat inherited conditions stemming from a single genetic mutation, including hemophilia B, have predominantly focused on the delivery of a working, or functional, gene using a viral vector.

Hemophilia B is an appropriate target for treatment with gene therapy because it is caused by a mutation of a single gene, the F9 gene, which is small enough that it can be packaged into an adeno-associated viral (AAV) vector.

How HEMGENIX gene therapy for hemophilia B works

HEMGENIX uses a gene therapy approach called gene transfer. Gene transfer therapy for hemophilia B starts by developing a package of genetic instructions—the functional, or working, gene. Then AAV vectors are created, which will eventually enter targeted liver cells. The package of genetic instructions is loaded into an AAV vector shell, acting as a delivery truck. Through a single IV infusion, the delivery truck heads toward the liver with its package.

Once delivered into the liver cells, the package of instructions is unloaded, enabling the liver to start generating factor IX, with the goal of allowing a person to produce their own elevated and protective levels of factor IX. After delivering its package, the AAV vector shell is broken down and eliminated. However, the genetic instructions remain to continue producing factor IX.

Built on science you can trust

Gene therapy is built on decades of clinical research. The first patients received gene therapy in 1970, and there are more than 250 AAV-based clinical trials currently underway across a variety of conditions. So not only is gene therapy with HEMGENIX EMHa great fit for hemophilia B, it’s based on years of scientific research.

Interested in learning more about the science behind HEMGENIX, a one-time infusion that offers years of consistent bleed protection? Explore all that gene therapy might offer for people with hemophilia B today!

*HEMGENIX was studied in a clinical trial of 54 male adults with moderately severe or severe hemophilia B. All people in the trial were taking factor IX prophy to treat their hemophilia B and were observed for at least 6 months on prophy before receiving HEMGENIX.

†AAV5, adeno-associated viral vector serotype 5.

IMPORTANT SAFETY INFORMATION

What is HEMGENIX?

HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:

  • Currently use Factor IX prophylaxis therapy, or
  • Have current or historical life-threatening bleeding, or
  • Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is administered as a single intravenous infusion and can be administered only once.

What medical testing can I expect to be given before and after administration of HEMGENIX?

To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.

HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.

What were the most common side effects of HEMGENIX in clinical trials?

In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.

What should I watch for during infusion with HEMGENIX?

Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.

What should I avoid after receiving HEMGENIX?

Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.

Please see full prescribing information [LINK TO: https://labeling.cslbehring.com/PI/US/Hemgenix/EN/Hemgenix-Prescribing-Information.pdf] for HEMGENIX.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch [LINK TO: www.fda.gov/medwatch], or call 1-800-FDA-1088.

You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.

HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC. HEMGENIX® is a registered trademark of CSL Behring LLC.

©2023 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA

www.CSLBehring.com USA-HGX-0464-MAY23

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