(Bloomberg) -- Pfizer Inc.’s gene therapy for a severe bleeding disorder met its goal in a pivotal late-stage trial, paving the way for the company to enter what’s proven to be a challenging market for drug companies.
The therapy performed better than a common treatment used by hemophilia A patients, known as Factor VIII replacement, in a measure of bleeding rates through at least 15 months of follow-up, Pfizer said Wednesday in a statement. The company said it will discuss the data with regulators in the coming months.
The results could provide a boost to Sangamo Therapeutics Inc., which developed the treatment in collaboration with Pfizer and has struggled with liquidity issues.
Sangamo shares rose 20% in trading before US markets opened, while Pfizer’s were little changed.
The therapy has faced a bumpy development path. At one point, the trial was put on hold when some patients’ levels of Factor VIII, a protein needed for blood clotting that’s addressed by the gene therapy, rose too high. The issues were later resolved and the trial was restarted.
Some drug companies have had a hard time gaining traction with new gene therapies, particularly in the hemophilia market. There are at least a dozen approved treatments in the US for hemophilia A, according to research from TD Cowen, including Roche Holding AG’s Hemlibra — a blockbuster with an annual price tag of more than $600,000. Another gene therapy for hemophilia A developed by BioMarin Pharmaceutical Inc. has been a commercial disappointment.
The disease causes people to bleed for longer than normal because they lack Factor VIII. A lifelong condition requiring constant monitoring and therapy, it occurs in about 25 of every 100,000 live male births worldwide.
Pfizer has been looking for new drugs as sales of its Covid medications plummet, while the shares trade at about half the level of their late 2021 peak of $61.25. The company in April won US approval for a gene therapy for hemophilia B, a different class of the bleeding disorder.
Its hemophilia A therapy aims to help patients produce Factor VIII themselves for an extended period of time, eliminating years of costly treatments and the burden of regular IV infusions or injections. Pfizer plans to file for approval for the treatment, called giroctocogene fitelparvovec, in early 2025, Sangamo executives said in a May earnings call.
(Updates with shares in fourth paragraph.)
©2024 Bloomberg L.P.
