Givinostat at a glance
Medicine class
Histone deacetylase inhibitor
Licensed use
Duchenne muscular dystrophy in people aged 6 years and older, irrespective of genetic variant
Form
Oral suspension measured with the supplied oral syringe
Administration
Take with food and shake thoroughly before measuring
Key monitoring
Blood counts, triglycerides and, for people at cardiac risk, ECGs
What is Givinostat used for?
The UK product is licensed for:
Duchenne muscular dystrophy in patients aged 6 years and older.
The indication is not restricted to a particular dystrophin-gene variant.
How does Givinostat work?
Givinostat blocks histone deacetylase enzymes, which influence the expression of genes involved in muscle repair and related processes. This may reduce inflammation, scarring and fatty replacement while supporting muscle regeneration, although its precise mechanism in Duchenne muscular dystrophy remains uncertain.
Givinostat preparations and strengths
Oral suspension
Route: Oral
Strengths: 8.86 mg/mL
How to take Givinostat
Use only the supplied oral syringe and follow the specialist team's instructions.
Take the suspension with food.
Shake the closed bottle thoroughly until the liquid looks uniform.
Measure with the supplied syringe and use immediately; do not store suspension in a filled syringe.
The person taking it should sit upright while the suspension is slowly given towards the inside of the cheek.
Wash the syringe with water after use and let it dry.
Givinostat side effects
Common or expected effects
- Diarrhoea
- Nausea or vomiting
- Abdominal pain
- Reduced platelet count
- Raised triglycerides
- Fever
- Constipation
- Reduced appetite
- Tiredness
- Muscle or joint pain
- Rash
Get urgent medical advice
- Bleeding or marked bruising associated with reduced platelets
- Severe or persistent diarrhoea or vomiting, particularly with dehydration
- Fainting, collapse or symptoms of a significant abnormal heart rhythm
Givinostat in pregnancy
Givinostat should not be used during pregnancy. Human pregnancy data are unavailable and animal studies found reproductive effects. Anyone who becomes pregnant while taking it should contact the prescribing specialist immediately.
Givinostat while breastfeeding
Givinostat should not be used while breastfeeding because it is unknown whether it enters human milk and a risk to the child cannot be excluded. Discuss alternatives with the specialist team.
Givinostat interactions
Give the specialist and pharmacist a complete medicines list. Important interactions include:
Medicines that prolong the QT interval
Examples include amiodarone, sotalol, ondansetron, azithromycin, clarithromycin, ciprofloxacin, fluconazole, risperidone and some anaesthetics. These can add to the risk of an abnormal heart rhythm.
Sensitive oral CYP3A4 substrates
Givinostat weakly inhibits intestinal CYP3A4, so medicines including tacrolimus, sirolimus, simvastatin, sildenafil or midazolam may require closer toxicity monitoring.
OCT2 substrates
Exposure to sensitive medicines such as metformin or dofetilide may increase because givinostat weakly inhibits OCT2.
Common questions about Givinostat
Answers are fully visible for fast scanning and source review.
Does Givinostat work for every Duchenne genetic variant?
Its UK indication is not limited to a particular genetic variant, although individual suitability requires specialist assessment.
Does Givinostat cure Duchenne muscular dystrophy?
No. Evidence indicates that it can slow functional decline, but function still worsened over time in both the givinostat and placebo groups.
Why are blood tests needed?
Givinostat can lower platelets and other blood-cell counts and raise triglycerides. Testing helps identify clinically important changes.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.