Mitapivat for sickle cell disease: FDA decision status
Agios has asked the US FDA to approve mitapivat, already approved for pyruvate kinase deficiency and thalassemia, for sickle cell disease.
Status: pending FDA decision
FDA goal date (PDUFA): . Sickle cell disease is not yet an approved use of mitapivat in the US.
Last checked: . The FDA may act on or before the goal date, and the date can move if the review is extended. We will update this box after the FDA acts.
Mitapivat is not yet FDA-approved for sickle cell disease. Agios filed a supplemental application under the accelerated approval pathway, the FDA granted Priority Review, and the FDA goal date is 1 November 2026.
What mitapivat is
- Mitapivat is an oral activator of the enzyme pyruvate kinase, developed by Agios Pharmaceuticals. [1][3]
- It is already approved in the US as Pyrukynd for pyruvate kinase deficiency and as Aqvesme for thalassemia. [1] See our Pyrukynd page.
- The pending application is a supplemental New Drug Application (sNDA) for sickle cell disease, filed under the FDA accelerated approval pathway and granted Priority Review. [1][2]
- The brand name for a sickle cell approval has not been announced in the sources we checked. It will be confirmed from the FDA label.
What the main trial showed
The application rests on RISE UP, a Phase 2/3 trial in people with sickle cell disease aged 16 and older. [3][4] In the Phase 3 part, 40.6% of patients on mitapivat reached the hemoglobin response target, compared with 2.9% on placebo (p<0.0001). The second primary endpoint, the yearly rate of sickle cell pain crises, did not reach statistical significance. [3] Agios has started REIGNITE, a Phase 3 confirmatory trial. [1]
Who it may be for
The application covers people with sickle cell disease. The exact approved age range and patient group will be set by the FDA label if it is approved. Until then, the treating hematologist decides whether a patient might be a candidate.
Mitapivat in Saudi Arabia and the UAE
Mitapivat is not new to the Gulf. On 17 October 2026, Pyrukynd (mitapivat) appeared in the Saudi SFDA drugs list, and Pyrukynd had active codes in the Abu Dhabi DoH drug code list (release 26 September 2026). [5][6] According to Agios, mitapivat is approved for adults with thalassemia in Saudi Arabia and the UAE, and Agios has filed for approval in sickle cell disease in Saudi Arabia. [1] We found no report of a Gulf approval for sickle cell disease as of 17 October 2026.
How Gulf patients may access it
- Before a US decision: use for sickle cell disease is through clinical trials, or through a developer access program if one exists. We have not confirmed whether Agios runs one for sickle cell disease; the treating physician can ask Agios directly.
- After a US approval: where the medicine or this use is not yet approved locally, the treating physician can usually apply to the national regulator to import it for a named patient, with a prescription, a medical report and a justification. The regulator decides. Use outside a locally approved indication is always a decision for the physician and the regulator.
- Our role: Reserve Meds coordinates access to FDA-approved medicines. We help families organise documents, work with the treating physician and arrange compliant logistics after approval. Price: quote on request. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.
Ask about mitapivat
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Frequently asked questions
Is mitapivat approved for sickle cell disease in the US?
Not yet. The FDA goal date for a decision is 1 November 2026. Until the FDA acts, sickle cell disease is not an approved use in the US.
Is mitapivat a new drug?
No. The same molecule is already approved in the US as Pyrukynd for pyruvate kinase deficiency and as Aqvesme for thalassemia. This application would add sickle cell disease.
What did the RISE UP trial show?
In the Phase 3 part, 40.6% of patients on mitapivat reached the hemoglobin response target versus 2.9% on placebo. The reduction in yearly pain crises did not reach statistical significance.
Is mitapivat available in Saudi Arabia or the UAE?
Pyrukynd (mitapivat) appeared in the SFDA drugs list and in the Abu Dhabi DoH drug code list on 17 October 2026, and Agios says it is approved for thalassemia in both countries. For sickle cell disease, Agios has filed in Saudi Arabia. Use for sickle cell disease before local approval is a decision for the physician and the regulator.
What does it cost?
Quote on request. We do not publish prices. Your coordination fee is quoted in writing for your case before you commit.
Sources
- Agios, Second Quarter 2026 financial results and business update, 30 Jul 2026 (accessed 17 Oct 2026)
- Agios, U.S. FDA Grants Priority Review to Agios' sNDA for Mitapivat in Sickle Cell Disease, 7 Jul 2026 (accessed 17 Oct 2026)
- Agios, RISE UP Phase 3 results at EHA 2026, 13 Jun 2026 (accessed 17 Oct 2026)
- ClinicalTrials.gov, NCT05031780 (RISE UP) (accessed 17 Oct 2026)
- Saudi Food and Drug Authority, Drugs List (Pyrukynd, mitapivat; checked 17 Oct 2026)
- Abu Dhabi DoH drug code list, release 26 Sep 2026 (Pyrukynd, active codes; checked 17 Oct 2026)
This page is general information, not medical advice. Treatment decisions belong to the patient's own physician and the relevant regulator. Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner.
Reviewed by the Reserve Meds clinical operations team. Last reviewed . Editorial standards.
Related: All pending FDA decisions · Pyrukynd (mitapivat) · US-approved, not yet registered in the Gulf