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On Heels of ASH Data, Pfizer Eyes FDA Submission for New Hemophilia Drug

Pfizer’s Experimental Drug for Hemophilia A and B Achieves Pivotal Study Goals

Pfizer Inc. has announced that its experimental drug for hemophilia A and B has met the goals of a pivotal study, reducing bleeding episodes. The company presented the full results of the study at the annual meeting of the American Society of Hematology, and is expecting to submit the results to regulatory authorities soon.

Hemophilia is a rare genetic disorder that affects the body’s ability to form blood clots. People with hemophilia A and B are at risk of excessive bleeding, even from minor injuries. The condition can cause serious complications, including joint damage, organ failure, and even death.

Pfizer’s experimental drug, PF-06741086, is a recombinant factor VIII (FVIII) therapy. It is designed to replace the missing clotting factor in people with hemophilia A, and to increase the levels of clotting factor in people with hemophilia B.

The pivotal study was a randomized, double-blind, placebo-controlled trial that included more than 200 patients with hemophilia A and B. The study evaluated the safety and efficacy of PF-06741086 compared to placebo.

The results of the study showed that PF-06741086 was effective in reducing bleeding episodes in both hemophilia A and B patients. In patients with hemophilia A, the drug reduced bleeding episodes by up to 95%. In patients with hemophilia B, the drug reduced bleeding episodes by up to 90%.

The study also showed that PF-06741086 was well-tolerated, with no serious adverse events reported. The most common side effects were injection site reactions and headache.

The results of the study are encouraging and suggest that PF-06741086 could be an effective treatment for hemophilia A and B. If approved, it could provide a much-needed treatment option for people with these conditions.

Pfizer is now preparing to submit the results of the study to regulatory authorities. If approved, PF-06741086 could become the first recombinant FVIII therapy for hemophilia A and B.

The results of the study are a major milestone for Pfizer and for people with hemophilia A and B. The drug could provide a much-needed treatment option for these patients, and could help improve their quality of life.

Pfizer is committed to developing innovative treatments for rare diseases, and the results of this study are a testament to that commitment. The company is hopeful that PF-06741086 will be approved and will be able to help people with hemophilia A and B.