FDA approves Jazz's Defitelio

FDA approved an NDA for Defitelio defibrotide from Jazz Pharmaceuticals plc (NASDAQ:JAZZ) to treat hepatic veno-occlusive disease (VOD) in patients with renal or pulmonary dysfunction after a hematopoietic stem cell transplant (HSCT). Defitelio is the first FDA-approved therapy for the indication.

Global head of R&D and CMO Karen Smith told BioCentury that Jazz expects to begin shipping Defitelio within a week at a wholesale acquisition cost (WAC) of $825 per vial. Dosing is about three vials per day for children and nine vials daily for adults, for a minimum of 21 days. Jazz said dosing may vary based on course of treatment. ...