Biotech
Wednesday, July 24th, 2024 5:59 pm EDT
Key Points
- Successful Gene Therapy Trial: Pfizer’s experimental gene therapy for hemophilia A has succeeded in a large late-stage trial, potentially setting the stage for its approval. This treatment could be Pfizer’s second gene therapy to enter the U.S. market, following Beqvez, which was approved for hemophilia B in April.
- Impact on Existing Treatments: The new therapy significantly reduced the number of bleeding episodes in patients with moderately severe to severe hemophilia A and outperformed the current standard treatment of routine infusions that replace the Factor VIII protein. Hemophilia A affects about 25 in every 100,000 male births globally.
- Market Competition and Challenges: Pfizer’s gene therapy will compete with BioMarin Pharmaceutical’s Roctavian, which has faced a slow market rollout since its U.S. approval last year. BioMarin is considering whether to divest its hemophilia A therapy, priced at $2.9 million, amid concerns about patient uptake and market dynamics.
Pfizer announced on Wednesday that its experimental gene therapy for hemophilia A has achieved success in a large late-stage clinical trial, positioning it for potential approval. This treatment targets a rare genetic blood-clotting disorder, hemophilia A, which is caused by a deficiency in the blood-clotting protein factor VIII. If approved, it would be Pfizer’s second gene therapy to enter the U.S. market, following the approval of Beqvez in April for hemophilia B, a different type of the bleeding disorder.
The therapy is being co-developed with Sangamo Therapeutics, whose stock surged by over 60% upon the release of the trial data, although it later retraced some of those gains. Pfizer’s stock remained relatively unchanged. This gene therapy represents a significant investment by Pfizer in the burgeoning field of gene and cell therapies, which aim to address the root cause of diseases with potentially curative or transformative effects. These therapies are seen as a potential replacement for traditional, ongoing treatments for chronic conditions.
Hemophilia A affects about 25 in every 100,000 male births globally, according to Pfizer. The condition impairs blood clotting, leading to a higher risk of spontaneous and severe bleeding. The new treatment has been shown to drastically reduce the number of annual bleeding episodes in patients with moderately severe to severe hemophilia A over a 15-month period, outperforming the existing standard of care, which involves regular infusions to replace the deficient factor VIII protein.
The study is ongoing, and Pfizer plans to present more data at upcoming medical conferences. If approved, Pfizer’s gene therapy will compete with BioMarin Pharmaceutical’s Roctavian, another one-time treatment for hemophilia A that has faced a slow market rollout since its approval in the U.S. last year. BioMarin is reportedly considering whether to divest its hemophilia A therapy, which is priced at $2.9 million. The competitive landscape for gene therapies in hemophilia A will be closely watched as the market develops.
For the full original article on CNBC, please click here: https://www.cnbc.com/2024/07/24/pfizers-gene-therapy-for-hemophilia-a-succeeds-in-late-stage-trial.html
