FDA extends review of mitapivat to February 1, 2027, for sickle cell disease treatment

Key Facts

Agios Pharmaceuticals set February 1, 2027, as the new target date for the U.S. Food and Drug Administration’s decision on its supplemental application for mitapivat to treat sickle cell disease, replacing the original November 1, 2026, date. The company said in a statement on October 30 that the agency extended the review by three months after classifying its response to a request for additional information as a major amendment. The application concerns mitapivat, which is marketed as PYRUKYND for a different indication: hemolytic anemia in adults with PK deficiency. The company submitted the application through the accelerated approval pathway and received priority review. The new date sets a specific target for a regulatory decision, but does not mean final approval for the new indication has been granted.

The extension process began when the FDA requested additional information on October 9 to help assess whether a risk evaluation and mitigation strategy, known as REMS, was needed. Agios submitted its response on October 19, including about nine months of updated safety data through October 9. On October 28, the agency told the company that the response constituted a major amendment to the application, adding three months to the review. The timeline shows that the extension was tied to review of the additional information and assessment of potential safety requirements. When the application was accepted for priority review, the company had said the target review timeline would be reduced from 10 months under the standard pathway to six months.

Agios said the additional follow-up data did not show liver injuries in patients with sickle cell disease of the same pattern and severity as those observed in mitapivat trials in adults with thalassemia. It added that the drug’s benefit-risk profile had not changed. The statement comes as the agency assesses whether REMS is needed and does not represent an FDA decision on the application. The company’s application is based on the phase 2 and phase 3 RISE UP trials. In phase 3, the study included 207 participants aged 16 years and older, randomly assigned in a 2-to-1 ratio to mitapivat or placebo.

The rate was 2.9% in the placebo group over the same period. These results are part of the application’s background, while the agency’s decision on the indication remains under review. Chief Executive Brian Goff said sickle cell disease affects about 100,000 people in the United States. The company added that it sees the potential indication as an important growth driver, an estimate attributed to the company that does not specify projected revenue or a regulatory outcome.

Under the accelerated approval pathway, the company began the phase 3 confirmatory REIGNITE trial in July 2026, after the first participant received a dose. The trial is required under the accelerated approval pathway and was designed to study reductions in transfusion burden among patients with sickle cell disease aged 12 years and older. Agios said it would continue label discussions with the FDA, including potential elements of a REMS program. It added: "We are ready to launch." The next regulatory milestones center on the new target date of February 1, 2027, and completing label discussions and potential safety requirements.