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

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?

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.

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

2. 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.

3. 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.

4. 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.

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.

©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

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

Back-to-school tips for kids and young adults with hemophilia

Sponsored by Sanofi

Entering a new grade or moving away to school can be nerve wracking for any student, but starting the school year with a rare bleeding disorder has its extra challenges. Finding the best way to manage your own care can help you feel more independent and confident in any new environment. Here’s how to help ease these periods of transition, from childhood to the teenage years and on into adulthood.

Childhood to teenage years
Teenage years bring a lot of changes, like becoming more in-the-know and involved with managing your own hemophilia care. In addition to the tips below, learn more from your care team about how hemophilia affects playing sports, puberty, and other activities.

• Learn the basics about your condition: Being open to learning about hemophilia from a young age can lead to more independence in the future. Participating in care can start early, too. Begin to gather materials and learn how to keep an environment clean. Once comfortable, take the next step in mixing factor and tracking infusions.
  
  
• Build healthy habits: A healthy weight can help protect joints, which is especially important if you have a bleeding disorder. Eating healthy and getting enough exercise are two great goals. Good nutrition and physical activity can help strengthen muscles and bones. Just be sure to talk to your doctor about bleeding risks and how to protect yourself before you try a new activity.

• Let the right people know: School nurses and teachers should know about your bleeding disorder, since they’re the ones who can support you while you’re at school. A guidance counselor or social worker should know all about Individualized Education Programs (IEPs) that provide special services and instruction to students who need more support.

• Plan ahead when going away from home: Sleepovers and camps can be so much fun. But it’s important to be ready in case a bleed happens. To prepare, have factor, infusion supplies, medical identification, and an infusion log or app. Make sure emergency information and contacts are also up to date and easy to access.

Telling friends and classmates about a bleeding disorder is a matter of individual preference. A simple way of explaining the condition might sound like:

“I have hemophilia, which means my blood doesn’t act like most people’s blood. If I get hurt, like a bump or bruise, it takes me longer to stop bleeding—on the inside and outside. I take medicine to help it stop.”

Teenage years to young adulthood
With age comes a greater feeling of empowerment to take charge of hemophilia and make the care decisions that come with it. Along the way, be sure to lean on support from your family and care network. Your care team is an important resource for more on specific topics like oral care, sexual health and safety, disability rights, and insurance matters. Here are a few ways to prepare for college or moving away from home:

• Maintain healthy habits: Beyond good eating and workout routines, consistently seeing a provider is an important part of navigating life with hemophilia. There may be a need to find new providers when moving away from home or a need to find an adult care provider. Be sure to get regular physical exams and checkups, and always follow your doctor’s prescribed dosing schedule. It’s also good to learn where to get bleeding disorder supplies and the location of the nearest HTC in your area.

• Get to know the community: Moving away from home can sometimes feel like an isolating experience. Getting involved in community organizations and volunteering may introduce you to new opportunities to meet people, find hobbies, and establish a support network. Your Sanofi CoRe or a HTC can help you find a hemophilia chapter nearby.

• Let others know: When living with roommates for the first time, starting a relationship, or talking to classmates, it may be important for them to be made aware of your hemophilia and know what to do in the case of an emergency. An example of telling someone that you have hemophilia might sound like this:
  
  “I have hemophilia. That means that my blood doesn’t clot the same as other people’s, which can lead to prolonged bleeds above and below my skin. It’s a genetic condition that is manageable with treatment.”

Sanofi is committed to empowering people with hemophilia. If you’d like to learn more about navigating different life stages with hemophilia, you can download our Through the Stages guide. You can also connect with your local Sanofi Community Relations and Education, or CoRe, Manager, for additional education and resources.

MAT-US-2305870-v1.0-07/2023