mitapivat
Orphan DrugFDA Approved (2022), EMA Approved (2022)
Description
Mitapivat is a first-in-class oral pyruvate kinase (PK) activator for hemolytic anemia due to pyruvate kinase deficiency (PKD). It enhances the activity of deficient PK enzymes, improving red blood cell survival and reducing hemolysis. This represents a significant advance for a disease previously managed only with transfusions and splenectomy.
Indications & Therapeutic Use
Hemolytic anemia due to pyruvate kinase (PK) deficiency in adults
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
mitapivat
| Generic Name | mitapivat |
| Brands | 1 brand available |
| Active Ingredient | mitapivat sulfate |
| Drug Class | Hemolytic anemia due to pyruvate kinase (PK) deficiency in adults |
| Manufacturer | Agios Pharmaceuticals |
| Dosage Forms | Oral tablet, 5mg, 20mg, 50mg |
| Medical Code | B06AX02 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 10 days |
| Reg. Status | FDA Approved (2022), EMA Approved (2022) |
| Clinical Trial | NCT03853798 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes