Why is Hemgenix the most expensive drug in the world?

Gene therapy treatments like Hemgenix are extremely expensive because they often require complicated and advanced drug design and manufacturing techniques, may be used to treat a rare disease with fewer patients, and often have a much higher success rate than older treatments, leading to fewer long-term, expensive therapies, which may justify the cost in some instances.

Hemgenix allows people living with hemophilia B to produce their own factor IX and can lower the risk of bleeding. It is administered as a single intravenous (IV) infusion and is given only once to introduce a working copy of the Factor IX gene into the body.

Costs associated with design and manufacturing of innovative gene therapies like Hemgenix can be much more expensive than traditional pharmaceuticals, like pills or capsules. Gene therapies are often single-dose, injected treatments involving a complicated manufacturing process that may take years of research to perfect and gain approval by the FDA.

In addition, gene therapies may lead to a higher rate of success and result in less ongoing medical and drug therapy; therefore, the cost may be considered justifiable. Typically, patients with hemophilia B have to adhere to strict, expensive, lifelong infusion schedules of factor IX, a blood clotting factor.

Gene therapies may also be approved to treat rare diseases, considered by the FDA to be a condition that impacts fewer than 200,000 people in the U.S. Because there are fewer people who will receive treatment, the manufacturers may need to increase the cost to help cover research and development expenses (to help make a profit), and in turn fund further research.

How much is Hemgenix?

Hemgenix is used to treat hemophilia B, a rare, lifelong bleeding disorder. The single dose therapy is priced at $3.5 million, according to CSL Behring, which made it the most expensive drug at the time of FDA approval in November 2022.

Manufacturers of drugs for rare diseases often have financial and insurance support teams that can help you and your doctor navigate treatment initiation. You can contact HEMGENIX Connect at 1-833-436-0021 Monday through Friday, 8 AM-8 PM ET.

What is Hemgenix used for?

Hemgenix (etranacogene dezaparvovec-drlb) is a one-time gene therapy for adults with hemophilia B. It allows people living with hemophilia B to produce their own factor IX and can lower the risk of bleeding with a single dose.

• Hemgenix is approved to be used in adults with hemophilia B who currently use factor IX therapy to help slow bleeding, have life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.
• Hemophilia B is caused by a single gene defect. It results in missing or insufficient levels of blood clotting Factor IX, a liver protein that helps to form blood clots.
• People with the condition are particularly vulnerable to bleeding in their joints, muscles, and internal organs (including the brain), leading to pain, swelling, and joint damage. Other symptoms include heavy bleeding after surgery, an injury, or a dental procedure. Some patients may have bleeding episodes without a clear cause.

How does Hemgenix work?

Hemgenix works by supplying a noninfectious viral vector (AAV5) to carry genetic DNA instructions to the liver, where factor IX proteins are then generated. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA.

The treatment introduces a new working F9 gene into the body which provides instructions to allow people with hemophilia B to make their own factor IX.

Does Hemgenix cure hemophilia B?

Studies have shown Hemgenix can dramatically reduce the rates of bleeding, but people treated with this medicine will still have the mutation that causes hemophilia B.

In the ongoing HOPE-B clinical trial, Hemgenix reduced the rate of annual bleeds and 94% of patients discontinued factor IX infusions. Seven to 18 months after Hemgenix, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54%.

Studies provided by the manufacturer have shown that a positive treatment effect can last for up to at least 2 years. According to CSL Bering, clinical studies monitoring its effects to 5 years are currently in progress.

The most common side effects (incidence ≥5%) were liver enzyme elevations, headache, elevated levels of blood creatine kinase, flu-like symptoms, infusion-related reactions, fatigue, nausea and generally feeling unwell.

This is not all the information you need to know about Hemgenix for safe and effective use and does not take the place of your doctor’s directions. Review the full product information and discuss this information and any questions you have with your doctor or other health care provider.