(BPT) - By Brandpoint

Michael was only four years old when he was diagnosed with hemophilia B, a genetic bleeding disorder that affects the body's ability to generate factor IX, a protein crucial for blood clotting. "It was scary in the beginning," Michael said. "Kids that age like to copy stuff on TV, and I wanted to play sports or be like Spider-Man. As a preschooler, I really had no idea how to comprehend my diagnosis. And then to learn that I'd have to stick myself with needles multiple times a month made me anxious."

Hemophilia B causes prolonged bleeding and increases the risk for internal bleeding in the muscles and joints, which can be life threatening if untreated. Missing or insufficient amounts of factor IX can be replaced by medicine to help the body clot properly. The standard treatment for people with hemophilia B involves lifelong, preventative or on-demand factor replacement therapy, which may be infused multiple times a month intravenously (into a person's veins). However, recent advancements in gene therapy offer new hope.

HEMGENIX^® (etranacogene dezaparvovec-drlb) provides a one-time infusion that can elevate factor IX levels for years, offering greater bleed protection and significantly reducing the need for regular infusions. Michael, who received HEMGENIX, shares his journey with this innovative treatment.

This is Michael's personal experience. Every patient experience is different. For more information, visit https://www.hemgenix.com.

Q: Before you tried gene therapy, you were on prophylaxis treatment. What was that like?

A: I had to learn to self-infuse prophylaxis from a very early age. Otherwise, I'd have to go to the hospital for treatment anytime I got injured - even from something small like jamming my finger playing basketball.

Once I was consistent with my infusions, I didn't need to go to the hospital as often, but I still sometimes experienced breakthrough bleeds. I tried not to let hemophilia B hold me back, and I participated in equestrian classes, swimming, basketball and golf, though I had to prepare for activities more than the average kid. I knew if I was going to play basketball, I may get injured, so I would self-infuse before I left the house. I also had to have my medicine with me in case I got hurt or I had to go to the hospital. It was just something I had to deal with.

Q: How did you learn about gene therapy? What motivated you to move forward with treatment?

A: My condition meant I was in and out of the doctor's office a lot, so I became pretty close to my care team. While I was in college a few years ago, they introduced me to a new treatment option called HEMGENIX.

Around the same time, I had suffered a pretty serious injury, tearing one of my pectoral muscles at the gym. I had to undergo surgery, which was scary because my hemophilia puts me at a higher risk of bleeding during the surgery and recovery. I had to self-infuse more frequently during this time and relied more on my family than ever as an adult. I thought, if there's a chance to stop sticking myself and help me become more independent, I'll take it. After discussing my options with my doctor, we decided that HEMGENIX would be a good option for me.

Q: What was receiving gene therapy like?

A: After going through the pre-screening tests and getting approval from my insurance company, I received an infusion date. HEMGENIX was quite new at the time, and there wasn't a treatment center near my home, so my grandmother and I drove from Louisiana to Mississippi for my infusion. I remember being very excited the entire drive. After having to self-infuse prophylaxis multiple times a month for most of my life, I was hopeful to receive a one-time treatment that could potentially reduce or eliminate my regular infusions for years.

The infusion process took a little over an hour, followed by a few hours of monitoring for infusion-related reactions before I could go home. For me, it was a simple process, and I used the time to get homework and studying done while at the center. Even though I knew what to expect, I still couldn't believe how easy it was for me.

Q: What's life like now after your infusion?

A: After receiving HEMGENIX, there were several follow-up appointments I had to do with my doctor, including liver and blood enzyme tests to monitor for any elevations. I also had to watch for any side effects such as headaches, flu-like symptoms, fatigue, nausea, or feeling unwell. Today, my life no longer revolves around my infusion schedule. I haven't experienced a breakthrough bleed since receiving HEMGENIX and I no longer need regular infusions. I've noticed that I feel more confident and less stressed because I know my factor IX levels are steady. I'm not always thinking about if and when I need to infuse. I exercise and play basketball now without having to self-infuse beforehand or afterwards.

I'm currently focused on pursuing my MBA, which I am excited to complete soon. From there, I'm going to pursue a career in business, and I look forward to many of life's new adventures without the stress of constant infusions.

Q: What message would you like to share with the broader community about living with hemophilia B?

A: I've been managing hemophilia B since elementary school and while I tried not to let it stop me from doing the things I wanted, it definitely can be physically and mentally challenging at times. It was always a nagging thought in the back of my head - something I had to constantly consider and be aware of. Having a support system in place is very important.

When my doctor introduced me to HEMGENIX, I was thrilled by the potential benefits but also nervous. Learning all I could about the benefits and risks ultimately helped me make the best decision for myself. However, everyone is different, and needs can change over time, so it's great that we have so many choices available today. I encourage everyone living with hemophilia B to regularly discuss their treatment options with their doctor to find what works best for them.

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.

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