The U.S. Food and Drug Administration has designated as an orphan drug an Alexion Pharmaceuticals’ long-acting antibody that may be used to treat a rare blood disorder.
The so-called ALXN1210 biologic developed by the New Haven-based firm is an antibody being evaluated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, life-threatening blood disorder in which a patient’s red blood cells are destroyed.
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S.
The status provides the developer with benefits and incentives, including a period of marketing exclusivity if the drug receives regulatory approval.
