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New conditionally approved treatment for patients with Duchenne muscular dystrophy (DMD)

January 10, 2025

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the conditional marketing authorization of givinostat (Duvyzat), a medication used to treat Duchenne muscular dystrophy (DMD). Givinostat is a nonsteroidal drug indicated for the treatment of patients aged 6 years of age and older with all genetic variants of DMD.

DMD is a muscle wasting condition that causes progressive muscle weakness. It usually only affects boys and those assigned male at birth. It’s caused by alterations in a protein called dystrophin. This causes muscle fibres to break down. They’re replaced by fibrous or fatty tissues that cause the muscle to gradually weaken. Around 100 boys are born with DMD each year, and there are about 2,500 people living with the condition in the UK at any one time.

Givinostat comes as an oral suspension to be taken twice daily. It is a drug from a family of molecules called HDAC inhibitors (histone deacetylase) which reduce in­flammation and fibrosis in muscle tissues. HDAC inhibitors also promote muscle regeneration and slow down the progression of DMD.

This approval is supported by evidence from a randomized, double-blind, placebo-controlled 18-month study involving 179 ambulatory patients with DMD who were given a placebo or givinostat for 18 months.

The most common side effects of the medicine (which may affect more than 1 in 10 people) include abdominal pain, decrease in blood platelet count (which can increase risk of bleeding), diarrhea, elevated levels of triglycerides (a type of blood fats), fever, nausea and vomiting. The prescribing information for givinostat advises healthcare providers to assess patients’ platelet counts and triglyceride levels prior to initiating treatment. During treatment, regular monitoring of platelet counts and triglycerides is recommended to determine whether dosage adjustments are necessary.  Additionally, givinostat may lead to QTc prolongation, a condition where the heart takes longer than normal to recharge between beats, which can elevate the risk of irregular heart rhythms. Patients with certain heart conditions or those taking other medications that prolong QTc intervals should avoid using givinostat.

As with any medicine, the MHRA will keep the safety and effectiveness of givinostat under close review.  Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.

Learn more: Givinostat conditionally approved to treat patients with Duchenne muscular dystrophy (DMD) - GOV.UK

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