Mitapivat for Sickle cell disease
The FDA is expected to decide on Mitapivat for Sickle cell disease by November 1, 2026, under a sNDA from Agios Pharmaceuticals.
What is being decided
Agios Pharmaceuticals has a sNDA under FDA review for Mitapivat in Sickle cell disease. Oral pill under priority review for accelerated approval; would be the first pyruvate kinase activator for sickle cell disease. The filing rests on the placebo-controlled RISE UP Phase 2 and Phase 3 trials in patients 16 and older. In Phase 3, mitapivat met its hemoglobin response endpoint, but its other primary endpoint, reducing sickle cell pain crises, did not reach statistical significance. The confirmatory REIGNITE trial is testing whether it reduces transfusion needs. Mitapivat is already approved for PK deficiency (Pyrukynd, 2022) and thalassemia (Aqvesme, 2025).
Who this decision matters to
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. This leads to chronic pain, organ damage, and life-threatening crises. Gene therapy and newer disease-modifying drugs are transforming outcomes.
Prevalence: About 100,000 Americans with SCD; 1 in 13 African-American births; 1 in 36 Hispanic-American births. See our full Sickle Cell Disease page for current treatments, recruiting trials, and community resources.
About the drug
Mitapivat: first-in-class oral allosteric activator of pyruvate kinase-R (PKR) that increases ATP production in red blood cells to reduce hemolysis. Our full Mitapivat profile covers how it works, side effects in plain language, and its trial history.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Sickle Cell Disease treatment stands today
Sickle cell disease treatment is advancing dramatically with gene therapy successes and newer disease-modifying approaches including fetal hemoglobin induction therapies. Base editing and other novel genetic approaches are entering human testing. The Sickle Cell Disease Association of America connects patients with trials. In December 2023 the FDA approved the first 2 cell-based gene therapies for sickle cell disease: exagamglogene autotemcel (Casgevy), the first FDA-approved treatment to use CRISPR gene editing, and lovotibeglogene autotemcel (Lyfgenia). Both are one-time treatments made from the patient's own blood stem cells, and both require high-dose chemotherapy (myeloablative conditioning) before the infusion. Casgevy is approved for people 2 and older with recurrent vaso-occlusive crises (pain crises), expanded from 12 and older in July 2026, and Lyfgenia is approved for people 12 and older with a history of vaso-occlusive events. Gene therapy offers potential cure for eligible patients, while newer medications can significantly reduce vaso-occlusive crises. Your hematologist can discuss which therapies and trials match your disease severity and medical history. Early consultation with comprehensive sickle cell centers optimizes both current management and future trial participation. Genetic counseling helps families understand inheritance and family screening.
Meanwhile, 213 Sickle Cell Disease trials are recruiting
Whatever the FDA decides here, research on Sickle Cell Disease does not stop. A few currently enrolling studies, US sites first:
Other drugs Trial Friend tracks for Sickle Cell Disease
Get notified when new sickle-cell-disease trials open or existing trials change status, add sites, or update eligibility.
Our coverage
Frequently asked questions
When will the FDA decide on Mitapivat?
The FDA's target decision date (PDUFA date) for Mitapivat in Sickle cell disease is November 1, 2026, disclosed by Agios Pharmaceuticals. The agency can act before this date and occasionally runs past it.
What is Mitapivat being reviewed for?
Agios Pharmaceuticals submitted a sNDA for Mitapivat in Sickle cell disease. Oral pill under priority review for accelerated approval; would be the first pyruvate kinase activator for sickle cell disease. The filing rests on the placebo-controlled RISE UP Phase 2 and Phase 3 trials in patients 16 and older. In Phase 3, mitapivat met its hemoglobin response endpoint, but its other primary endpoint, reducing sickle cell pain crises, did not reach statistical significance. The confirmatory REIGNITE trial is testing whether it reduces transfusion needs. Mitapivat is already approved for PK deficiency (Pyrukynd, 2022) and thalassemia (Aqvesme, 2025).
What happens after the Mitapivat decision?
If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from Agios press release. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.