FDA Delays PDUFA Action on Agios’ Mitapivat sNDA for SCD
The FDA has delayed a decision on Agios Pharmaceuticals’ supplemental New Drug Application (sNDA) seeking additional approval for mitapivat in sickle cell disease (SCD) until next year. The post FDA Delays PDUFA Action on Agios’ Mitapivat sNDA for SCD appeared first on GEN - Genetic Engineering and Biotechnology News .
The FDA has delayed a decision on Agios Pharmaceuticals' supplemental New Drug Application (sNDA) seeking additional approval for the drug mitapivat in sickle cell disease (SCD) until February 1, 2027. The delay was triggered by a major amendment to the sNDA, requiring additional information to assess the need for a Risk Evaluation and Mitigation Strategy (REMS).
Agios submitted updated safety data from the ongoing open-label extension periods of the Phase II/III RISE UP trial, showing a hemoglobin response in 41% of patients compared to 3% on placebo. Serious adverse events were reported in 20% of patients on mitapivat versus 29% on placebo. Despite the delay, Agios remains confident in mitapivat's potential to transform care for people with sickle cell disease, as the drug has a confirmed risk-benefit profile and management expects no additional delays.
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