The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome, a rare and life-threatening disease that affects the liver and has no approved pharmacologic treatment option.
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview of Dravet syndrome, a severe form of epilepsy that is part of a group of diseases known as SCN1A-related seizure disorders.
The FDA granted Olaparib (Lynparza) an orphan drug designation by for the treatment of patients with pancreatic cancer,
Len Walt, Vice President, Medical Affairs, North America discusses his company’s focus on rare diseases, specificially MPS IIIA or Sanfilippo A syndrome.
The FDA approved Revcovi (elapegademase-lvlr) injection in the United States, for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
The FDA expanded the approval of Hemlibra (emicizumab-kxwh) to include routine prophylaxis to prevent or reduce the frequency of bleeding episodes among individuals with hemophilia A without factor VIII inhibitors.