Pfizer's gene therapy for hemophilia A (PFE.US) achieved its goal in a crucial late-stage trial, paving the way for the company to enter this challenging market.
Pfizer's gene therapy for hemophilia A (PFE.US) achieved its goal in a crucial late-stage trial, paving the way for the company to enter this challenging market.
Pfizer said in a statement on Wednesday that in at least 15 months of follow-up, the therapy outperformed factor VIII replacement therapy in terms of bleeding indicators for patients with hemophilia A. The company said it would discuss these data with regulatory agencies in the coming months.
This result may boost Sangamo Therapeutics (SGMO.US), which is facing liquidity issues and is collaborating with Pfizer to develop this drug.
Some pharmaceutical companies are facing challenges in promoting new gene therapies, especially in the field of hemophilia treatment. According to TD Cowen's research, there are at least a dozen drugs approved in the United States for the treatment of hemophilia A, including Roche's Hemlibra, a heavyweight product with annual sales of over $600 million. Another gene therapy for the treatment of hemophilia A developed by Biomarin Pharmaceutical (BMRN.US) is disappointing in terms of commercialization.
Due to a lack of factor VIII, patients with hemophilia A have a longer bleeding time than normal people. This is a lifelong disease that requires continuous monitoring and treatment, affecting approximately 25 out of every 100,000 live male births worldwide.
As its sales of COVID-19 drugs plummet, Pfizer has been searching for new drugs, and its stock price is currently about half of its peak of $61.25 at the end of 2021. In April of this year, the company received U.S. approval for a gene therapy for hemophilia B.
Pfizer's hemophilia A therapy aims to help patients produce factor VIII on their own for a long time, eliminating the burden of expensive treatment and regular intravenous infusions or injections. Sangamo executives said on the company's May earnings call that Pfizer plans to apply for approval of this therapy, called giroctocogene fitelparvovec, in early 2025.
As of press time, Pfizer's stock price fell 0.31%, and Sangamo Therapeutics' stock price rose 1.92% before the opening.