Aisha Khan, PhD
Legal and Regulatory Affairs Committee: North America
University of Miami
United States
The U.S. Food and Drug Administration (FDA) approved Hemgenix (AMT-061, etranacogene) gene therapy for the treatment of adults with hemophilia B (congenital factor 9 deficiency). Hemophilia is a disorder in which a person’s blood does not clot normally, which can lead to spontaneous or excessive bleeding, according to the Centers for Disease Control and Prevention (CDC). [1]
Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life, said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a press release “Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia,” he said. [2]
Clinical trial data suggest that a single dose of Hemgenix will provide patient with moderate to severe hemophilia with adequate protection from uncontrolled bleeding for eight years, and potentially longer. Investigators conducted two studies of 57 adult men with severe or moderately severe hemophilia B. Effectiveness was established based on decreases in the men’s annualized bleeding rate. In one study with 54 participants, the subjects had increases in Factor IX activity levels, a decreased need for routine Factor IX replacement prophylaxis, and a 54% reduction in annualized bleeding rate compared with baseline.
Many physicians think that treatment can completely change a patient’s life in many ways if they have a successful outcome with this therapy.
1. Will Be Able to Receive Hemgenix?
Hemgenix is indicated for adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening bleeding or have repeated, serious Who spontaneous bleeding episodes. Before Hemgenix is administered, the candidate is tested for factor IX inhibitors; if they test positive, they are not eligible for the therapy. Currently, patients receive frequent IVs of a protein that helps blood clot and prevent bleeding.
2. How Much Will Hemgenix Cost?
The list price of Hemgenix is set at $3.5 million for one time treatment. According to Nuijten, the price makes Hemgenix the most expensive medicine in the world. [3] The drug is about 66.7% more expensive than Zolgensma, which was previously the most expensive drug in the world at $2.1 million per treatment course. Zolgensma was approved by the FDA in 2019 for the treatment of spinal muscular atrophy.
Does this price justify cost savings for the overall healthcare system and lowering the economic burden of hemophilia B by reducing annual bleed rates? Would this high price tag make it difficult for public health systems to finance the therapy? In the United States, would this treatment be available to common public without an extremely comprehensive health insurance coverage? However, at present, it is not known how durable and affordable the treatment will be. We will know in 5 to 10 years.
Updates and regulatory news
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OTAT Director Bryan, who has worked at the FDA for more than 20 years, will depart by the end of March.
https://pink.pharmaintelligence.informa.com/PS147654/FDAs-Cell-Gene-Therapy-Office-Head-Wilson-Bryan-Set-To-Retire
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References:
- Centers for Disease Control and Prevention - About CDC’s Work on Hemophilia. Last reviewed by August 19, 2022.
https://www.cdc.gov/ncbddd/hemophilia/aboutus.html
- US Food and Drug Administration - FDA Approves First Gene Therapy to Treat Adults with Hemophilia B. Released by November 22, 2022.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b?utm_medium=email&utm_source=govdelivery
- Nuijten M. Pricing Zolgensma - the world's most expensive drug. J Mark Access Health Policy. 2021 Dec 29;10(1):2022353. doi: 10.1080/20016689.2021.2022353. PMID: 34992762; PMCID: PMC8725676.
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