Givinostat
DUVYZAT
What it isHistone deacetylase inhibitor; Duchenne muscular dystrophy.
Why this oneIt targets fibrosis and inflammation downstream of dystrophin loss, so eligibility does not depend on the mutation.
What limits itThrombocytopenia, triglyceride elevation, and diarrhea limit treatment; monitor platelets and lipids, and review QT interactions.
FDA-approved for
- Duchenne muscular dystrophy (2024)
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Duchenne muscular dystrophy | Patients ≥6 yr | Weight-based: 22.2 mg BID (10–<20 kg) up to 53.2 mg BID (≥60 kg) | 53.2 mg BID |
Renal Undefined
Hepatic Undefined
Instructions
- Administer orally twice daily with food
- Obtain baseline platelet count and triglycerides; do not start if platelets <150 x10^9/L
- Modify dose for thrombocytopenia, diarrhea, hypertriglyceridemia, or QTc prolongation
Cautions
- Hematological changes: thrombocytopenia, anemia, neutropenia
- Increased triglycerides
- GI disturbances (diarrhea)
- QTc prolongation — avoid in patients at risk for ventricular arrhythmia
Adverse reactions
- Diarrhea
- Abdominal pain
- Thrombocytopenia
- Nausea/vomiting
- Hypertriglyceridemia
- Pyrexia
Pregnancy No human data; animal studies show fetal harm (decreased fetal weight, variations, embryofetal/offspring mortality).
Lactation No human or animal data.
NO BOXED WARNINGS
Principal riskThrombocytopenia
Cost, est. cash$50,000–$65,000/month
Generic entry2038 est.
Classes and tags
Clinical profile
Legacy pregnancy categoryNot assigned — PLLR-era drug
Defining liabilityGenerally tolerated at therapeutic doses; clinically important risks are agent-specific and increase with interactions or organ impairment.
Mechanism of action: Givinostat ballicule
