Givinostat conditionally approved to treat patients with 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.

Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:

Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.

We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.

As with all products, we will keep its safety under close review.

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. Further data is required for the efficacy in non-ambulatory patients and will be subject to review following completion of additional clinical trials, as detailed in the condition associated with approval.

The study found that participants treated with givinostat over an 18-month period took significantly less time to climb four stairs compared to those given placebo.

A secondary efficacy endpoint was the change from baseline to month 18 in physical function as assessed by the North Star Ambulatory Assessment (NSAA) — a scale commonly used to rate the motor function in boys with DMD who are capable of walking. Compared to placebo, patients treated with givinostat saw less worsening in their NSAA score after 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.

ENDS

Notes to editors 

1. The new conditional marketing authorisation was granted on 20 December 2024 to Italfarmaco S.p.A. More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.

2. Giovinostat has been conditionally approved through the International Recognition Procedure (IRP). A conditional marketing authorisation is an early temporary license in which we may accept less completed clinical studies than would be necessary to issue a full marketing authorisation, provided the manufacturer clearly indicates when complete clinical data will be available. However, CMA post-approval conditions are determined on a case-by-case basis, and don’t have to be limited to providing further clinical data. A CMA lasts for one year and can be renewed annually.

3. For more information about Duchenne muscular dystrophy (DMD), visit: https://www.nhs.uk/conditions/muscular-dystrophy/

4. The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.

5. The MHRA is an executive agency of the Department of Health and Social Care.

6. For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.