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Pfizer U.S. drugmaker ABC said Wednesday its experimental gene therapy for a rare genetic blood clotting disorder has succeeded in a large, late-stage trial, paving the way for possible approval.
The hemophilia A treatment could become the company's second gene therapy to enter the U.S. market after Pikvis, which was approved in April to treat a less common type of the bleeding disorder called hemophilia B.
Pfizer is partnering with Sangamo TherapeuticsPfizer shares rose more than 60% on Wednesday after the data was released. Pfizer shares were flat on Wednesday.
Pfizer is among a number of drugmakers investing in the fast-growing field of gene and cell therapies — expensive treatments that target a patient’s genetic or cellular source to cure or significantly alter the course of a disease. Some health experts in the industry expect these therapies to replace the traditional lifelong medications patients take to manage chronic conditions.
Hemophilia A is a lifelong disease caused by a deficiency of a blood-clotting protein called factor VIII. Without enough of this protein, blood can’t clot properly, increasing the risk of spontaneous bleeding and severe bleeding after surgery. The condition occurs in about 25 out of every 100,000 male births worldwide, Pfizer said in a statement, citing data.
Pfizer said its one-time treatment significantly reduced the number of annual bleeding episodes in patients with moderate to severe hemophilia A after 15 months. The company said the drug worked better than the current standard treatment for the disease, which consists of routine injections that replace the factor VIII protein.
“For people living with hemophilia A, the physical and emotional impact of having to prevent and treat bleeding episodes with frequent intravenous infusions or injections cannot be understated,” Dr. Andrew Levat, the trial’s lead investigator, said in a statement.
Pfizer said the study is ongoing and will present additional data at upcoming medical meetings.
If approved, Pfizer's treatment would compete with Biomarin Pharmaceuticals CompanyBioMarin's treatment has been delayed since it won U.S. approval last year, raising questions about how many patients would take Pfizer's drug if it entered the market.
BioMarin is reportedly considering withdrawing its $2.9 million hemophilia A treatment.
