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Agios Anticipates PK Deficiency Diagnosis Ramp-Up After Pyrukynd Approval

Reimbursement Challenges

 
• By Alaric DeArment

The company hopes to grow the current diagnostic rate from 30% to 70% over two to five years. “Optimal reimbursement” for mitapivat will likely take a year, with Medicare/Medicaid lagging.

3-D Rendering Red Blood Cells
The FDA approved Agios' Pyrukynd for pyruvate kinase deficiency on 17 February • Source: Shutterstock

Agios Pharmaceuticals, Inc. has already been working on increasing screening for rare hereditary anemias, and now that the US Food and Drug Administration has approved its Pyrukynd (mitapivat) for patients with pyruvate kinase (PK) deficiency, the company is trying to more than double the rate of patients currently being diagnosed.

The FDA approved the drug on 17 February for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, based on the pivotal Phase III ACTIVATE trial as well as the ACTIVATE-T trial in patients regularly receiving blood transfusions

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