Access to treatments

DMD treatment Givinostat reaches important stage in European approval process

Givinostat, a new treatment for Duchenne muscular dystrophy (DMD), has reached an important stage in the European Union approval process.

Givinostat has had its Marketing Authorization Application (MAA) to the European Medicine Agency (EMA) validated. This means that the EMA can begin to review it.

If approved, Givinostat will be available for eligible DMD patients within the European Union.

Background to submission

Givinostat is a histone deacetylase (HDAC) inhibitor that is produced by the Italian pharmaceutical company Italfarmaco.

The MAA submission was based on the safety and efficacy results from the phase three EPIDYS clinical trial which tested Givinostat in ambulant patients with DMD.

The phase three evaluation of Givinostat demonstrated that it can slow DMD progression in boys six years and older.

Priority review in the US

This submission comes just two months after the US Food and Drug Administration (FDA) granted priority review for Givinostat, which is expected to be completed by the end of the year.

If approved by the FDA, it would be available for eligible DMD patients in the US.

What it means for people living with DMD in the UK

This is good news for the DMD community in the UK.

If Givinostat is approved by the EMA, Italfarmaco can use this approval to seek a quicker approval by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK.

In the UK, currently, companies can take advantage of the European Commission Decision Reliance Procedure, which has been put in place to support the MHRA speeding up its review of treatments approved by the EMA. This will be replaced by the new International Recognition Procedure from the start of 2024.

While neither lead to automatic approval of treatments approved by the EMA, they can support reducing timelines for MHRA approval, as the agency can rely on the EMA feedback and opinion to inform their decision.

We will keep you updated with any new developments with Givinostat’s review process in the UK and globally.

Published on 6 September 2023

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