A once-daily, extended-release form of the amphetamine and dextroamphetamine mixture Adderall (Adderall XR, Shire) has been approved for treatment of attention-deficit/hyperactivity disorder. The new product is supplied in capsules that provide double-pulsed amphetamine delivery, prolonging the amphetamine release compared with immediate-release Adderall.
FDA Approves Pfizer’s Gene Therapy for Beqvez for Hemophilia
April 26th 2024Beqvez (fidanacogene elaparvovec) is priced at $3.5 million, which is on parity with Hemgenix, the first one-time therapy to treat adults with hemophilia B. Pfizer’s warranty will refund insurers and continue to provide coverage for patients if they change insurers.
FDA Issues Complete Response Letter for Pz-Cel to Treat Epidermolysis Bullosa
April 22nd 2024Prademagene zamikeracel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information on manufacturing practices.