Agios Pharmaceuticals,
Inc. AGIO, a leader in the fields of cancer metabolism and rare
genetic disorders of metabolism, today announced that the United States Food
and Drug Administration (FDA) has granted the company orphan drug designation
for its investigational medicine AG-348 for the treatment of pyruvate kinase
(PK) deficiency, a rare form of hemolytic anemia. AG-348, a first-in-class,
orally available activator of pyruvate kinase-R (PKR) enzymes, met its primary
endpoints in two Phase 1 healthy volunteer studies – a single ascending dose
study and multiple ascending dose study. In addition, data presented in
December 2014 at the 54^th Annual Meeting of the American Society of
Hematology (ASH) provided early proof-of-mechanism for AG-348. Based on these
findings, Agios plans to initiate a Phase 2 clinical trial in patients with PK
deficiency in the first half of 2015.
"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," said Chris Bowden, M.D., chief medical officer of
Agios. "PK deficiency can result in lifelong medical problems and is an
example of our focus on underserved diseases with significant medical needs."
The FDA's Office of Orphan Drug Products grants orphan status to support
development of medicines for underserved patient populations, or rare
disorders that affect fewer than 200,000 people in the U.S. This designation
provides certain benefits, including market exclusivity upon regulatory
approval, if received, exemption of FDA application fees and tax credits for
qualified clinical trials.
Market News and Data brought to you by Benzinga APIs© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Comments
Loading...
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in