FDA approved sapropterin on December 13, 2007, to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due ro BH4-responsive phenylketonuria (PKU).
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.