Last reviewed 2026-04-23 · How we verify

Etavopivat tablets

Etavopivat activates the protein kinase PKC (protein kinase C) to increase fetal hemoglobin (HbF) production, thereby reducing sickling and hemolysis in red blood cells.

Etavopivat activates the protein kinase PKC (protein kinase C) to increase fetal hemoglobin (HbF) production, thereby reducing sickling and hemolysis in red blood cells. Used for Sickle cell disease.

At a glance

Mechanism of action

Etavopivat is a PKC activator that promotes the expression of fetal hemoglobin in erythrocytes. By increasing HbF levels, the drug reduces polymerization of sickle hemoglobin and improves red blood cell survival, thereby decreasing vaso-occlusive crises and hemolytic anemia in patients with sickle cell disease.

Approved indications

• Sickle cell disease

Common side effects

• Headache
• Diarrhea
• Nausea
• Abdominal pain

Key clinical trials

• A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease (PHASE2)
• A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants (PHASE1)
• A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease (PHASE2)
• A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) (PHASE3)
• A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke (PHASE2)

Primary sources

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