Pyrukynd (mitapivat)
- Region
- 🇪🇺 EU
- Active substance
- mitapivat
- Company
- Agios Netherlands B.V.
- Status
- Authorised
- Therapeutic area
- Genetic Diseases, Inborn; Anemia, Hemolytic
- CHMP opinion
- Thu 15 Sep 2022
- EU approval
- Wed 09 Nov 2022
- Designations
- Orphan, Additional monitoring
- Application
- EMEA/H/C/005540
- ATC
- B06AX04
- Official record
- www.ema.europa.eu
Indication
Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients (see section 4.4).
Regulatory milestones
PYRUKYND: PDUFA target action date (non–transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia)
Agios Pharmaceuticals, Inc. (AGIO); mitapivat; sNDA; status: extended; "…PDUFA goal date for PYRUKYND U.S. sNDA in thalassemia set for December 7, 2025 • CHMP adopted positive opinion for PYRUKYND in thalassemia; EC decision expected by early 2026 • RISE UP Phase 3 trial topline results in sickle cell disease by year-end; potential U.S."; filed 2025-10-30 · source
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.