Pfizer Inc. (NYSE:PFE) and Sangamo Therapeutics Inc (NASDAQ:SGMO) revealed that they observed favorable findings from their collaborative early-stage gene therapy study that seeks to address severe hemophilia A.
The study, which was dubbed the Phase 1/2 Alta study, involved a developmental gene therapy called giroctocogene fitelparvovec (SB-525, or PF-07055480) aimed at treating severe hemophilia A. The latter is the rare blood condition where the blood fails to clot properly. The early-stage clinical trial involved five patients suffering from the same condition, and all of them were treated with a 3e13 vg/kg dose of the gene therapy.
The results of the clinical trial indicate that the patients had a sustainable level of the vital protein that is needed for proper clotting for as long as 14 months. Sangamo and Pfizer have been collaboratively developing the new treatment. However, they are just two of the numerous companies that are also trying to come up with a viable treatment for severe hemophilia A.
Fortunately for the duo, their pipeline gene therapy for the genetic clotting-related condition demonstrated significant progress. The findings from study revealed that the treatment managed to lower the episodes of patients bleeding due to the health condition by 90%. Pfizer and Sangamo have already submitted their findings to the U.S Food and Drug Administration (FDA), and they expect the regulatory authority to announce a favorable opinion by August 21.
The gene therapy might deliver much-needed relief to hemophilia A patients
Sangamo and Pfizer’s treatment is aimed at helping patients to produce the protein that facilitates proper clotting on their own. Chronic hemophilia A is a debilitating health condition in which patients experience painful bleeding in the joints and muscles. The condition can even result in severe joint damage over the long-term.
Results observed by Pfizer and Sangamo in their early-stage study will help get closer to a viable treatment for patients suffering from severe hemophilia A. A regulatory opinion from the FDA will likely lead to a green light for the two companies to advance their research on the treatment. This means that more clinical trials might be on the way soon.