The FDA today granted orphan drug designation to AG-348 for the treatment of pyruvate kinase deficiency, a rare form of hemolytic anemia, according to a press release from the drug’s manufacturer.
AG-348 (Agios Pharmaceuticals) is a first-in-class, orally available activator of pyruvate kinase-R enzymes. Currently, there is no approved or disease-modifying treatment for pyruvate kinase (PK) deficiency.
AG-348 met its primary endpoints in two phase 1 healthy volunteer studies, which included a single ascending dose study and a multiple ascending dose study, according to the release. In addition, data presented at the 2014 ASH Annual Meeting provided early proof-of-mechanism for AG-348.
"We are pleased to achieve another milestone in the clinical program for AG-348, the first medicine in development designed to treat the underlying cause of PK deficiency," Chris Bowden, MD, chief medical officer of Agios Pharmaceuticals, said in the press release. "PK deficiency can result in lifelong medical problems and is an example of our focus on underserved diseases with significant medical needs."
Agios Pharmaceuticals plans to initiate a phase 2 trial to further evaluate AG-348 in patients with PK deficiency in the first half of 2015.
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics 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 United States.