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# Mitapivat Moving Toward Accelerated Approval in Sickle Cell: Agios
- URL: https://www.fdaweb.com/mitapivat-moving-toward-accelerated-approval-in-sickle-cell-agios/
- Published: 2026-03-31T12:00:00.000Z
- Updated: 2026-09-14T13:36:28.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5160907

Agios Pharmaceuticals says it plans to seek accelerated approval for mitapivat in sickle cell disease, following a pre-submission meeting with FDA. At the meeting, FDA advised the company to include a confirmatory clinical trial as part of an accelerated approval pathway submission. Agios says it has already submitted a proposal for that trial and expects to file a supplemental new drug application (sNDA) in the coming months.

Mitapivat, an oral pyruvate kinase activator, is designed to improve red blood cell function by boosting cellular energy production and reducing levels of 2,3-diphosphoglycerate, a molecule associated with the sickling of red blood cells. The drug is currently approved to treat various types of rare inherited anemias by activating the enzyme pyruvate kinase.

The regulatory update follows discussions centered on data from the company’s RISE UP clinical program, which includes Phase 2 and Phase 3 studies in patients with sickle cell disease. In the pivotal Phase 3 trial, mitapivat met one of its two primary endpoints, demonstrating a statistically significant improvement in hemoglobin response compared with placebo, according to the company. However, the second primary endpoint — reduction in the annualized rate of pain crises — did not reach statistical significance.

Despite that mixed outcome, Agios says patients who achieved the hemoglobin response also experienced clinically meaningful benefits, including fewer pain crises, reduced hospitalizations, and improved fatigue.

Sickle cell disease is a rare inherited blood disorder marked by abnormal hemoglobin that causes red blood cells to become rigid and sickle-shaped. The condition leads to chronic anemia, painful vaso-occlusive crises, and progressive organ damage, contributing to significant morbidity and early mortality.