FDA Approves Pyrukynd, the First Disease-Modifying Treatment for Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency

The U.S Food and Drug Administration (FDA) has approved Pyrukynd (mitapivat), the first disease-modifying treatment for hemolytic anemia in adults with pyruvate kinase (PK) deficiency. Pyrukynd, developed by Agios Pharmaceuticals, is a PK activator and the first in this drug class. The company is also awaiting approval by the European Medicines Agency, and a decision is expected by the end of 2022.

PK deficiency is a rare genetic disorder in which the PK-R gene is mutated, leading to shortened red blood cell lifespan and chronic accelerated red blood cell destruction (hemolytic anemia). The disease can lead to serious complications, including pulmonary hypertension, gallstones, osteoporosis, enlarged spleen, and iron overload. Current therapies for PK deficiency include red blood cell transfusions and splenectomy, which are invasive and can be associated with serious risks.

Pyrukynd will be available as an oral tablet to be taken twice daily. Its approval was based on two pivotal phase 3 trials. The ACTIVATE study evaluated the drug’s safety and efficacy in 80 adults with PK deficiency who were not receiving regular blood transfusions. The ACTIVATE-T trial was conducted in 27 adults with PK deficiency who had received regular blood transfusions. In the ACTIVATE study, 40% more patients taking Pyrukynd had increased hemoglobin compared to those taking placebo. In the ACTIVATE-T study, 33% of patients taking Pyrukynd saw a reduction in transfusion burden. The most common adverse reactions in both studies included decreased estrone and estradiol levels in males, increased urate, back pain, and joint pain.

Agios plans to initiate two studies in pediatric patients with PK deficiency by midyear.

The company is offering access programs intended to mitigate out-of-pocket costs for Pyrukynd. These include a copay program for eligible commercially-insured patients and a Patient Assistance Program for qualified uninsured and underinsured individuals. Eligible patients may potentially have access to this drug at no charge through one of these offers.