Credit: Shutterstock. Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes. The Food and Drug Administration (FDA) has accepted for Priority ...

Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...

Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare ...

(RTTNews) - Agios Pharmaceuticals Inc. (AGIO), said on Friday that the FDA has approved Pyrukynd for the treatment of hemolytic anemia in adults with pyruvate kinase or PK deficiency, a rare, ...