Biotech
Friday, April 26th, 2024 4:48 pm EDT
Key Points
- FDA approves Pfizer’s gene therapy for hemophilia B, named Beqvez, marking the company’s first gene therapy clearance in the U.S.
- Beqvez targets adults with moderate to severe hemophilia B and aims to enable patients to produce factor IX to prevent and control bleeding, offering a one-time treatment option superior to standard infusions.
- Priced at $3.5 million before insurance and rebates, Beqvez competes with CSL Behring’s Hemgenix in the market, posing potential challenges due to high costs and logistical issues despite its transformative potential for appropriate patients.
The Food and Drug Administration (FDA) approved Pfizer’s treatment for a rare genetic bleeding disorder, marking the company’s first gene therapy clearance in the U.S. Known as Beqvez, the drug is sanctioned for adults with moderate to severe hemophilia B who meet specific criteria. Despite the milestone, Beqvez comes with a substantial price tag of $3.5 million before insurance and rebates, making it one of the costliest drugs in the country. Hemophilia B, affecting over 7,000 individuals in the U.S., results from inadequate levels of a protein necessary for blood clotting, leading to prolonged bleeding and bruising. Beqvez is a one-time therapy designed to enable patients to produce the required protein themselves, thus mitigating bleeding episodes and reducing treatment burdens associated with standard infusions.
Clinical trials demonstrated Beqvez’s superiority over conventional hemophilia B treatment, which often necessitates frequent protein infusions. Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, highlighted the potential transformative impact of Pfizer’s drug, emphasizing its ability to alleviate medical and treatment burdens over the long term. For Pfizer, this approval represents a significant stride, particularly amid efforts to rebound from the downturn in its COVID-19 business. The company aims to bolster its portfolio with cancer drugs and therapies targeting other diseases.
Pfizer’s venture into gene and cell therapies aligns with broader industry trends, with such treatments poised to revolutionize disease management. Acquiring the rights to Beqvez from Spark Therapeutics in 2014, Pfizer is offering a warranty program to payers, providing financial protection against efficacy failure. Beqvez enters a competitive landscape, facing off against CSL Behring’s Hemgenix, another FDA-approved hemophilia B therapy. However, uptake challenges, including high costs and logistical issues, have tempered expectations for similar gene therapies. Looking ahead, Pfizer eyes FDA approval for marstacimab, an experimental antibody targeting hemophilia A and B, alongside ongoing developments in Duchenne muscular dystrophy gene therapy.
For the full original article on CNBC, please click here: https://www.cnbc.com/2024/04/26/fda-approves-pfizer-gene-therapy-beqvez-for-treatment-of-hemophilia-b.html
