Orphan Drugs Lead Specialty Medication FDA Approvals

February 1, 2023

Syringe with glass vials and medications pills drug

Specialty medications currently lead the charts in terms of new drug approvals by the US Food and Drug Administration (FDA). On top of the heap are orphan drugs, treatments for rare diseases or those with no current therapeutics. In a new Pharmacy Practice News article, learn more about the orphan drugs in the approval pipeline for 2023.

According to Marie Rosenthal, “Three products, one for hemophilia B and two for hemophilia A, are expecting approval: concizumab (Novo Nordisk), efanesoctocog alfa (Sanofi) and valoctocogene roxaparvovec (Roctavian, BioMarin). All of them have received an orphan drug designation. Hemophilia A and B affect 30,000 Americans, mostly men and boys, who produce little or no clotting factors. Most patients have hemophilia A (about 80%), which is caused by a deficiency in factor VIII; hemophilia B is caused by a factor IX deficiency. About 15% of patients develop inhibitors, which makes hemostasis more difficult to achieve.”

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