Histone deacetylase (HDAC) inhibitor Pregnancy: DMD is a disease predominantly of young male patients, so there are no adequate data to assess use in pregnant women. In animal studies, oral givinostat during organogenesis reduced fetal body weight and increased structural variations, and administration during pregnancy and lactation increased embryofetal and offspring mortality and caused neurobehavioural changes in the offspring.

Givinostat

Brand names: Duvyzat

Givinostat is an oral histone deacetylase (HDAC) inhibitor used in the treatment of Duchenne muscular dystrophy.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: Body-weight banded (patients 6 years of age and older): 10 kg to less than 20 kg — 22.2 mg twice daily (2.5 mL); 20 kg to less than 40 kg — 31 mg twice daily (3.5 mL); 40 kg to less than 60 kg — 44.3 mg twice daily (5 mL); 60 kg or more — 53.2 mg twice daily (6 mL). Volumes refer to the 8.86 mg/mL oral suspension.

Route: Oral suspension, taken with food; measure the prescribed volume with the graduated oral syringe provided

Frequency: Twice daily with food

Max: 53.2 mg twice daily — described in the label as the maximum recommended human dose

INDICATION: Duchenne muscular dystrophy (DMD) in patients 6 years of age and older; the same weight-band table covers adults and children 6 years and older. BEFORE INITIATION: obtain and evaluate baseline platelet counts and triglycerides; do NOT initiate if the platelet count is less than 150 x 10^9/L. In patients with underlying cardiac disease or taking concomitant QT-prolonging medicines, obtain ECGs when initiating treatment, during concomitant use, and as clinically indicated. DOSE MODIFICATIONS for decreased platelets, diarrhoea or increased triglycerides (consider treatment interruption first) — FIRST reduction: 10 to less than 20 kg 17.7 mg twice daily (2 mL); 20 to less than 40 kg 22.2 mg twice daily (2.5 mL); 40 to less than 60 kg 31 mg twice daily (3.5 mL); 60 kg or more 39.9 mg twice daily (4.5 mL). SECOND reduction: 13.3 mg (1.5 mL), 17.7 mg (2 mL), 26.6 mg (3 mL) and 35.4 mg (4 mL) twice daily respectively. If the adverse reaction persists after the second reduction, discontinue. QTc: withhold if the QTc interval is greater than 500 ms or the change from baseline is greater than 60 ms. ADMINISTRATION: shake the suspension for at least 30 seconds by inverting the bottle 180 degrees and check homogeneity before each use. MISSED DOSE: do not take double or extra doses. PAEDIATRIC: safety and effectiveness below 6 years of age have not been established; the weight bands above are the label's dosing from 6 years and must be verified against a children's formulary before use. SOURCE LIMITATION: no UK SPC (eMC) was fetched in this bundle — this draft is distilled from the US DUVYZAT prescribing information and must be verified against UK labelling.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

None stated — the US label section 4 Contraindications reads 'None'

Side effects

Diarrhoea — most common adverse reaction (10% or more of treated patients); adjust dosage for moderate or severe diarrhoea
Abdominal pain and nausea/vomiting (10% or more)
Thrombocytopenia — dose-related; occurred in 33% of treated patients versus none on placebo, with maximum platelet decrease within the first 2 months and low counts sustained; led to dose reduction in 28%. Other myelosuppression (decreased haemoglobin, neutropenia) can occur
Hypertriglyceridaemia (10% or more)
Pyrexia (10% or more)
QTc prolongation — avoid use in patients at increased risk for ventricular arrhythmias

Interactions

Orally administered sensitive CYP3A4 substrates — givinostat is a weak intestinal CYP3A4 inhibitor; monitor closely where a small change in substrate plasma concentration may lead to serious toxicities
Sensitive substrates of the OCT2 renal uptake transporter — givinostat is a weak OCT2 inhibitor; monitor closely where a small change in substrate plasma concentration may lead to serious toxicities
Other products with a known potential to prolong the QTc interval — avoid concomitant use; if it cannot be avoided, obtain ECGs when initiating, during concomitant use and as clinically indicated

Clinical monograph

How it works

It inhibits histone deacetylase enzymes, modulating gene expression to reduce muscle inflammation, necrosis and fibrosis associated with dystrophin deficiency.

Prescribing in practice

It can prolong the QT interval and cause thrombocytopenia, so baseline and periodic ECG and platelet counts are required with dose adjustment as needed.
Treatment is initiated and supervised by specialists in neuromuscular disorders.
Gastrointestinal effects, hypertriglyceridaemia and reductions in blood counts can occur and may require dose modification.

Monitoring

Monitor full blood count (especially platelets), triglycerides and ECG (QT interval) at baseline and periodically.

Counselling the patient

Attend all blood tests and heart tracings as scheduled.
Report palpitations, fainting, unusual bruising or bleeding promptly.
Do not change or stop the medicine without specialist advice.

Evidence & guidelines

Benefit in Duchenne muscular dystrophy was supported by the EPIDYS randomised placebo-controlled trial.

Reference: SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Calculators

Pathways