Mitapivat for Sickle cell disease
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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 adults with PK deficiency (Pyrukynd, 2022) and adults with thalassemia (Aqvesme, December 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; about 1 in 365 Black or African American births and 1 in 16,300 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
Read our full Sickle Cell Disease treatment summary
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.
The Sickle Cell Disease page has every treatment and trial →Meanwhile, 198 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:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Sickle Cell Disease. Unsubscribe anytime.
Our coverage
Frequently asked questions
When will the FDA decide on Mitapivat?
The FDA's target date (PDUFA date) for Mitapivat in Sickle cell disease is November 1, 2026.
Who disclosed it: Agios Pharmaceuticals. The FDA does not publish these dates.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
Source: Agios press release
What is Mitapivat being reviewed for?
Agios Pharmaceuticals submitted a sNDA for Mitapivat in Sickle cell disease.
What to know: 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 adults with PK deficiency (Pyrukynd, 2022) and adults with thalassemia (Aqvesme, December 2025).
How it works: First-in-class oral allosteric activator of pyruvate kinase-R (PKR) that increases ATP production in red blood cells to reduce hemolysis.
More: the Mitapivat drug page explains it in plain language.
Is there already an FDA-approved treatment for Sickle Cell Disease?
Yes. Trial Friend's drug list includes 5 FDA-approved drugs for Sickle Cell Disease.
Approved: Droxia (hydroxyurea), Endari (l-glutamine), Adakveo (crizanlizumab), Casgevy (exagamglogene autotemcel), Lyfgenia (lovotibeglogene autotemcel).
Check: the Sickle Cell Disease page also searches FDA drug labels for approved treatments.
What did the Mitapivat trials show?
The ENERGIZE Phase 3 trial enrolled 194 patients with non-transfusion-dependent alpha or beta-thalassemia. 42% of patients on mitapivat achieved a hemoglobin increase of at least 1.0 g/dL sustained over weeks 12-24, compared to just 2% on placebo.
More: the Mitapivat drug page has the full trial history and sources.
What are the side effects of Mitapivat?
Aqvesme has a boxed warning for serious liver injury (hepatocellular injury). In the thalassemia trials, 5 patients had liver injury within the first 6 months and 2 needed hospital care; it improved after the drug was stopped.
More: the Mitapivat drug page lists the warnings in full.
Can I get Mitapivat before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 198 Sickle Cell Disease trials are recruiting now. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask Agios Pharmaceuticals directly. Registered programs are listed on ClinicalTrials.gov.
How much will Mitapivat cost?
Agios Pharmaceuticals has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Mitapivat decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
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.