Prior to February 2022, there were no approved disease-modifying therapies for those with pyruvate kinase (PK) deficiency. However, this has recently changed! According to a news release from biopharmaceutical company Agios Pharmaceuticals, Inc. (“Agios”), the U.S. FDA has approved PYRUKYND (mitapivat) for adults with hemolytic anemia caused by PK deficiency. Now, PYRUKYND stands as the first treatment of its kind and fills a huge unmet need within this community.

PYRUKYND Approval

So what exactly is PYRUKYND? This pyruvate kinase activator works by treating the defective enzyme and preventing red blood cells from breaking down too quickly. The treatment is orally administered. Altogether, following this approval, PYRUKYND should be available for patients by some time in March. Additionally, the European Medicines Agency is evaluating PYRUKYND and, if all goes well, could approve the treatment by the end of the year.

The U.S. approval hinged on data from the Phase 3 ACTIVATE and ACTIVATE-T clinical trials. In the first trial, 40% of those taking PYRUKYND saw increased hemoglobin levels; no patients who received a placebo saw similar results. Additionally, patients saw changes in hemolysis and erythropoiesis. Next, in the second trial, 33% of patients were able to reduce the burden of treatment, with 22% being able to remain transfusion-free. While PYRUKYND was relatively safe and well-tolerated, some adverse reactions did occur. These included:

• Jaundice (yellowing of the skin, eyes, and mucus membranes)
• Fatigue
• Shortness of breath
• Back and joint pain
• Atrial fibrillation

Moving forward, Agios also hopes to hold two additional clinical trials centered on evaluating PYRUKYND for children with PK deficiency.

Patient Support

Looking to learn more about PYRUKYND for PK deficiency? Want to connect with other patients and caregivers? Interested in learning more about financial assistance or support (including Patient Assistance Programs and $0 copays)?

Then look no further than myAgios Patient Support Services. Head to that link to learn more or enroll.

About Pyruvate Kinase (PK) Deficiency

Pyruvate kinase (PK) deficiency is a rare inherited condition which causes red blood cells to break down too quickly and easily (chronic hemolytic anemia). This condition results from PKLR gene mutations, which cause low levels of the pyruvate kinase enzyme. PK deficiency is incredibly variable; some patients may experience mild symptoms, while others may experience severe symptoms, complications, or interruptions to daily life. Associated symptoms include:

• Pallor (pale skin)
• Slowed growth
• Rapid heartbeat
• Recurrent gallstones
• Fast breathing
• Enlarged spleen
• Jaundice (yellowing of the skin, eyes, and mucus membranes)
• Fatigue and lethargy

Learn more about PK deficiency.