Pfizer on Wednesday said its experimental gene therapy for a rare genetic blood-clotting disorder succeeded in a large late-stage trial, paving the way for a potential approval.
Without enough of that protein, the blood cannot clot properly, increasing the risk of spontaneous bleeding and severe bleeding after surgery.
Pfizer said its one-time treatment significantly cut the number of annual bleeding episodes in patients with moderately severe to severe hemophilia A after 15 months.
If approved, Pfizer's therapy will compete with BioMarin Pharmaceutical 's one-time treatment Roctavian.
BioMarin is reportedly considering whether to divest its hemophilia A therapy, which costs $2.9 million.
Persons:
Andrew Leavitt, Pfizer
Organizations:
Pfizer, Sangamo Therapeutics, BioMarin
Locations:
U.S