FDA Approves Sanofi’s New Hemophilia Drug

The U.S. Food and Drug Administration (FDA) has approved Sanofi’s Qfitlia (fitusiran) for the treatment of Hemophilia.

Hemophilia is a genetic bleeding disorder caused by a dysfunction or deficiency of coagulation factor VIII or IX. Patients with this condition are unable to clot properly, leading to prolonged bleeding after injury or surgery. Qfitlia is an antithrombin-directed small interfering RNA (siRNA) therapy that reduces antithrombin levels, thereby increasing thrombin, an enzyme critical for blood clotting.

Qfitlia is administered u subcutaneously under the skin with a starting dose of once every two months and its dose and frequency are adjusted using INNOVANCE, Antithrombin companion diagnostic test. Qfitlia has advantages of lower frequency of dosing, very low volume and room temperature storage over other hemophilia drugs.

FDA had granted Qfitlia orphan drug status and fast track designation for approval.

FDA News Release on approval for Qfitlia

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