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hemgenix-logo-bg mobile-menu-bg HEMGENIX® (etranacogene dezaparvovec-drlb)

EXPLORING THE SCIENCE BEHIND GENE THERAPY FOR HEMOPHILIA B

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Patient portrayal; HEMGENIX not intended for women

therapy-gene

What is gene therapy?

Gene therapy uses advanced science to address the gene causing a condition. It either provides a working copy of the existing gene or turns off or changes the existing gene.

Gene therapies are of particular interest for the treatment of both hemophilia A and hemophilia B because each is caused by a single gene mutation in either the F8 or F9 gene, respectively. However, because the mutations are in different genes, hemophilia B cannot be treated with the same gene therapy used to treat hemophilia A.

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Hemophilia B is an appropriate target for treatment with gene therapy because:

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It is caused by a mutation of a single gene (the F9 gene)

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The F9 gene is small and can be packaged into an adeno-associated viral (AAV) vector

Here’s how gene transfer therapy works
for hemophilia B

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1
Working gene

The working gene

It starts by developing a package of genetic instructions—the functional, or working, gene

2
Delivery truck

The delivery vehicle

Then AAV vectors are created, which will eventually enter targeted liver cells

3
Bringing the working gene to the delivery truck

Bringing the two together

The package of genetic instructions is loaded into an AAV vector, acting as a delivery truck

4
Delivery of working gene

Special delivery

Through a single IV infusion, the delivery truck heads toward the liver with its package

5
Liver generating FIX

Generating their own factor IX

Once delivered into the liver cells, the package of instructions enables 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

6
Regenerating FIX in liver

Keep the factor IX going

After delivering its package, the AAV vector shell is broken down and eliminated. However, the genetic instructions remain to continue producing factor IX

Current unmet needs in hemophilia B

While there have been advances in hemophilia B treatment, the current standard of care for hemophilia B is lifelong infusions of factor IX replacement therapy. People who regularly infuse factor IX replacement products can still experience breakthrough bleeds, leading to joint damage, pain, and reduced quality of life.

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Joint damage

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Pain

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Reduced quality of life

People living with hemophilia B may benefit from a one-time infusion that offers consistent bleed protection lasting years instead of weeks

A recent CSL Behring–sponsored survey assessed the burdens of living with hemophilia B in 110 people who were receiving short-term prophylaxis or long-term prophylaxis.* It showed that:

Despite being on prophylaxis, those surveyed still experienced

an average of

4.2

spontaneous bleeds

over the past 6 months

The majority of people in the survey experienced joint damage and pain:

The majority of people in the survey experienced joint damage and pain:

65

%

had joint damage

The majority of people in the survey experienced joint damage and pain:

87

%

experienced joint pain at least a few times per month

*Short-term prophylaxis is prolonged treatment following a bleed until full recovery and prophylaxis prior to physical activity. Long-term prophylaxis is regular preventative injections. Of the 110 people surveyed, 29 were being treated with short-term prophylaxis only, 74 were being treated with long-term prophylaxis only, and 7 were being treated with both short-term and long-term prophylaxis.

Discover the possibilities of a one-time infusion

Elevated factor IX levels from gene therapy can offer:

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Ongoing prevention

Reduce risk of bleeding, including the risk of joint bleeds

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Freedom

Reduce or even eliminate the burdens of regular, long-term prophy

Redefine how you live with hemophilia B

See how HEMGENIX works

HEMGENIX is the first‑ever gene therapy for hemophilia B

See study results
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IMPORTANT SAFETY INFORMATION

Before you can receive HEMGENIX®, etranacogene dezaparvovec-drlb, your doctor will perform tests to check for Factor IX inhibitors. If you have inhibitors, or have had them in the past, you are not a candidate for HEMGENIX. Your doctor will also check your liver health and screen for hepatitis B and C, as active infection may affect how well the treatment works or increase the risk of side effects.

Infusion-related and allergic reactions (including anaphylaxis) can occur. You will be monitored throughout infusion and for at least 3 hours afterward. If a reaction occurs during infusion, the infusion may be slowed or interrupted as symptoms are addressed, then started at a slower rate once symptoms have resolved.

Liver effects including liver injury can occur. Your doctor will monitor your liver with blood tests every week for 3 months and then monthly up to 1 year after treatment. Increased liver enzymes may require corticosteroid treatment. Because HEMGENIX works in the liver, there is a potential (possible but not proven) risk of liver cancer, and some patients may be monitored for up to 5 years.

After treatment, your body will develop antibodies to HEMGENIX, which may affect how well it works.

Your doctor will monitor Factor IX levels and check for inhibitors. You may still need support to help control bleeding, especially in the initial weeks after treatment.

Most common side effects include increased liver enzymes, headache, increased blood creatine kinase, 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.

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.

INDICATION

HEMGENIX is indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who:

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

Please see full prescribing information for HEMGENIX.