Overview
Mitapivat is a novel, first-in-class pyruvate kinase activator. It works to increase the activity of erythrocyte pyruvate kinase, a key enzyme involved in the survival of red blood cells. Defects in the pyruvate kinase enzyme in various red blood cells disorders lead to the lack of energy production for red blood cells, leading to lifelong premature destruction of red blood cells or chronic hemolytic anemia. On February 17, 2022, the FDA approved mitapivat as the first disease-modifying treatment for hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, inherited disorder leading to lifelong hemolytic anemia. Mitapivat has also been investigated in other hereditary red blood cell disorders associated with hemolytic anemia, such as sickle cell disease and alpha- and beta-thalassemia.
Indication
Mitapivat is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
Associated Conditions
- Pyruvate Kinase Deficiency Anemia
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/08 | Not Applicable | Recruiting | |||
2024/10/18 | Phase 1 | Completed | |||
2024/10/07 | Phase 2 | Not yet recruiting | |||
2023/07/07 | Phase 2 | Not yet recruiting | |||
2023/03/21 | Phase 4 | ENROLLING_BY_INVITATION | |||
2022/11/09 | Phase 1 | Completed | |||
2022/01/03 | Phase 3 | Active, not recruiting | |||
2021/12/03 | Phase 3 | Active, not recruiting | |||
2021/09/02 | Phase 2 | Active, not recruiting | |||
2021/02/25 | Phase 3 | Active, not recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | 71334-220 | ORAL | 5 mg in 1 1 | 12/20/2023 | |
| Agios Pharmaceuticals, Inc. | 71334-205 | ORAL | 5 mg in 1 1 | 12/20/2023 | |
| Agios Pharmaceuticals, Inc. | 71334-215 | ORAL | 50 mg in 1 1 | 12/20/2023 | |
| Agios Pharmaceuticals, Inc. | 71334-210 | ORAL | 20 mg in 1 1 | 12/20/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Agios Netherlands B.V.,Zuidplein 36,1077XV Amsterdam,Netherlands | Authorised | 11/9/2022 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||
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