The U.S. Food and Drug Administration (FDA) has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. This marks a significant milestone as Pyrukynd is the first disease-modifying therapy approved for this rare, inherited blood disorder.
Clinical Trial Data
The FDA's approval was based on data from two Phase 3 clinical trials, ACTIVATE and ACTIVATE-T. The ACTIVATE trial, which included patients who were not regularly transfused, demonstrated a statistically significant increase in hemoglobin levels in 40% of patients treated with Pyrukynd, compared to 0% in the placebo group (p<0.0001). The ACTIVATE-T trial, involving regularly transfused patients, showed that 33% of patients achieved a significant reduction in transfusion burden, and 22% were transfusion-free during the 24-week fixed-dose period.
Hanny Al-Samkari, M.D., a hematologist and clinical investigator at Mass General Cancer Center and Harvard Medical School, noted, "The successful ACTIVATE and ACTIVATE-T studies demonstrate the impact of mitapivat in significantly improving hemolysis and anemia in PK deficiency."
Pricing and Access
Pyrukynd will be priced at approximately $334,880 per year. Agios is committed to providing robust patient access programs, including copay assistance and free medication for eligible patients, to ensure that the impact of Pyrukynd is maximized. Jackie Fouse, CEO of Agios, stated that the company is dedicated to making a difference for people with PK deficiency and expanding the reach of Pyrukynd to more patients with genetically defined diseases.
Ongoing and Future Studies
Agios is also planning to initiate two pivotal studies, ACTIVATE-kids and ACTIVATE-kidsT, in pediatric patients with PK deficiency in mid-2022. Furthermore, the company is advancing its Phase 3 ENERGIZE and ENERGIZE-T studies in adults with thalassemia, as well as its Phase 2/3 RISE UP study in sickle cell disease.
