Mitapivat

Generic Name: Mitapivat

Brand Names: Pyrukynd 5 Mg 4-week, Pyrukynd

Drug Type: Small Molecule

Chemical Formula: C_₂₄H_₂₆N_₄O_₃S

CAS Number: 1260075-17-9

Unique Ingredient Identifier: 2WTV10SIKH

Background: 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

Associated Therapies: -

Clinical Trials

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Phase 1

Active, not recruiting

Conditions: Healthy Participants

Interventions: Drug: Mitapivat
Drug: Midazolam

First Posted Date: 2024-10-18

Last Posted Date: 2024-11-19

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 20

Registration Number: NCT06648824

Locations: 🇺🇸
Fortrea Clinical Research Unit Inc., Dallas, Texas, United States

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

Phase 2

Not yet recruiting

Conditions: Sickle Cell Disease
Nephropathy

Interventions: Drug: Mitapivat

First Posted Date: 2024-02-29

Last Posted Date: 2024-11-14

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 40

Registration Number: NCT06286046

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Phase 4

Conditions: Anemia, Hemolytic
Pyruvate Kinase Deficiency

Interventions: Drug: Mitapivat

First Posted Date: 2023-03-21

Last Posted Date: 2024-12-24

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 6

Registration Number: NCT05777993

Locations: 🇨🇦
McMaster Centre for Transfusion Medicine McMaster University, Hamilton, Ontario, Canada
🇯🇵
Toho University - Omori Medical Center, Ota-Ku, Japan
🇯🇵
Tohoku University Hospital, Sendai, Miyagi, Japan

and more 1 locations

A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function

Phase 1

Completed

Conditions: Moderate Hepatic Impairment

Interventions: Drug: Mitapivat

First Posted Date: 2022-11-09

Last Posted Date: 2023-09-21

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 20

Registration Number: NCT05610657

Locations: 🇺🇸
Orlando Clinical Research Center (OCRC), Orlando, Florida, United States
🇺🇸
Orange County Research Center (OCRC), Tustin, California, United States
🇺🇸
Clinical Pharmacology of Miami (CPMI), Miami, Florida, United States

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

Phase 3

Active, not recruiting

Conditions: Pediatric Pyruvate Kinase Deficiency
Pediatric Hemolytic Anemia

Interventions: Drug: Mitapivat-matching placebo
Drug: Mitapivat

First Posted Date: 2022-01-03

Last Posted Date: 2024-12-18

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 30

Registration Number: NCT05175105

Locations: 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸
Children's Healthcare of Atlanta - Emory, Atlanta, Georgia, United States
🇺🇸
Stanford Medicine, Palo Alto, California, United States

and more 16 locations

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

Phase 3

Active, not recruiting

Conditions: Pediatric Hemolytic Anemia
Pediatric Pyruvate Kinase Deficiency

Interventions: Drug: Mitapivat
Drug: Mitapivat-matching placebo

First Posted Date: 2021-12-03

Last Posted Date: 2024-12-18

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 49

Registration Number: NCT05144256

Locations: 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇹🇷
Ege University Faculty of Medicine, Izmir, Adana, Turkey
🇺🇸
Weill Cornell Medical College, New York, New York, United States

and more 19 locations

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Phase 2

Active, not recruiting

Conditions: Sickle Cell Disease

Interventions: Drug: Mitapivat
Other: Mitapivat-matching placebo

First Posted Date: 2021-09-02

Last Posted Date: 2024-12-20

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 267

Registration Number: NCT05031780

Locations: 🇺🇸
University of California San Diego, La Jolla, California, United States
🇺🇸
UCLA Health, Los Angeles, California, United States
🇺🇸
Children's Hospital Oakland, Oakland, California, United States

and more 97 locations

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Phase 3

Active, not recruiting

Conditions: Transfusion-dependent Alpha-Thalassemia
Transfusion-dependent Beta-Thalassemia

Interventions: Drug: Placebo Matching Mitapivat
Drug: Mitapivat

First Posted Date: 2021-02-25

Last Posted Date: 2024-11-15

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 258

Registration Number: NCT04770779

Locations: 🇺🇸
Penn Medicine - University of Pennsylvania Health System, Philadelphia, Pennsylvania, United States
🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇮🇹
Ospedale Pediatrico Microcitemico, Cagliari, Italy

and more 75 locations

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Phase 3

Active, not recruiting

Conditions: Non-Transfusion-dependent Alpha-Thalassemia
Non-Transfusion-dependent Beta-Thalassemia

Interventions: Drug: Mitapivat
Drug: Placebo Matching Mitapivat

First Posted Date: 2021-02-25

Last Posted Date: 2024-07-11

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 194

Registration Number: NCT04770753

Locations: 🇧🇬
UMHAT "Sveti Georgi" EAD, Plovdiv, Bulgaria
🇧🇬
SHATHD Sofia, Sofia, Bulgaria
🇬🇷
Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School, Athens, Greece

and more 68 locations

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

Phase 1

Active, not recruiting

Conditions: Sickle Cell Disease
Hemolytic Anemia

Interventions: Drug: Mitapivat

First Posted Date: 2020-11-02

Last Posted Date: 2025-01-06

Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Target Recruit Count: 15

Registration Number: NCT04610866

Locations: 🇺🇸
National Institutes of Health Clinical Center, Bethesda, Maryland, United States

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