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FDA Approves Pyrukynd (mitapivat) for Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency

• The FDA has approved Pyrukynd (mitapivat) as the first disease-modifying therapy for hemolytic anemia in adults with pyruvate kinase (PK) deficiency. • Approval was based on Phase 3 ACTIVATE and ACTIVATE-T trials, which showed significant improvements in hemoglobin levels and reduced transfusion burden. • Pyrukynd, an oral PK activator, will be available with patient access programs, including copay assistance and free medication for eligible patients. • Agios plans to expand Pyrukynd's use to other blood disorders like thalassemia and sickle cell disease, with ongoing and planned clinical trials.

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.
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Reference News

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Agios scores FDA approval for PK deficiency drug Pyrukynd
ml.firstwordpharma.com · Feb 17, 2022

FDA approved Agios Pharmaceuticals' Pyrukynd for treating haemolytic anaemia in adults with PK deficiency, marking the f...

[2]
With drug approval, Agios hits first key milestone after major shakeup
biopharmadive.com · Feb 18, 2022

FDA approved Agios Pharmaceuticals' mitapivat (Pyrukynd) for pyruvate kinase deficiency, a rare anemia, marking a shift ...

[3]
Agios Announces FDA Approval of PYRUKYND® (mitapivat) as - GlobeNewswire
globenewswire.com · Feb 17, 2022

The FDA approved PYRUKYND® (mitapivat) for treating hemolytic anemia in adults with pyruvate kinase (PK) deficiency, bas...

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