PFE.US's A-type hemophilia gene therapy has achieved positive results in a key trial.
1min read Pfizer's (PFE.US) gene therapy for A hemophilia has met its primary endpoint in a key late-stage trial, paving the way for the company to enter this challenging market.
Pfizer announced in a statement Wednesday that its treatment was superior to the standard of care, which is Factor VIII replacement therapy, in bleeding rates in a follow-up of at least 15 months. The company said it would discuss the data with regulators in the coming months.
The result could boost Sangamo Therapeutics (SGMO.US), which is developing the drug with Pfizer and is facing liquidity issues. The company has faced challenges in marketing novel gene therapies, particularly in hemophilia treatments.
There are at least a dozen drugs approved for treating A hemophilia in the US, including Roche's Hemlibra, a blockbuster product with annual sales of more than $600m. Bayer's (BMRN.US) other gene therapy for A hemophilia has disappointed in the clinic.
A hemophilia A patient's blood does not clot because they lack Factor VIII, which causes their bleeding time to be longer than normal. It is a lifelong disease that requires continuous monitoring and treatment and affects about 1 in 400 male live births worldwide.
Pfizer has been looking for new medicines as its Covid drug sales have waned, and its shares are currently trading at about half their 2021 year-end closing price of $61.25. The company received US approval for a gene therapy for B hemophilia in April.
Pfizer's A hemophilia treatment aims to help patients produce Factor VIII for extended periods, eliminating the costly treatment and regular infusions or injections. Pfizer plans to apply for approval of the treatment, called giroctocogene fitelparvovec, in early 2025, executives said on a conference call in May.
As of the close, Pfizer shares were down 0.31 per cent and Sangamo Therapeutics shares were up 1.92 per cent.