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 FDA has extended its target action date under the Prescription Drug User Fee Act (PDUFA) by three months, from the originally scheduled November 1, 2026, to February 1, 2027, Agios acknowledged in a statement.
Investors punished Agios with a stock selloff that sent the company’s share price slumping nearly 18% from $37.23 to $30.55 on Friday. The three-month extension was triggered by what the FDA deemed a major amendment to the sNDA—namely the submission, at the agency’s request, of additional information to assess whether a Risk Evaluation and Mitigation Strategy (REMS) would be necessary for mitapivat in SCD.
Agios responded to the FDA on October 19 by submitting approximately nine months of updated safety data through October 9 from the ongoing open-label extension periods of the Phase II/III RISE UP trial (NCT05031780). According to data published October 24 in The Lancet, 56 (41%) of 138 patients in the mitapivat group showed a hemoglobin response, compared with two (3%) of 69 patients on placebo.
Source: genengnews.com · Image: Naitō Museum of Pharmaceutical Science and Industry 20210529 17 by 先従隗始, CC0, via Wikimedia Commons
Start your 7-day trial and see what the database can do →
