Access to treatments

New DMD drug shows success in Phase 3 clinical trial

Data published from placebo controlled clinical trial supports beneficial effect in DMD patients.

Italfarmaco Group announced yesterday positive top line data and statistical differences in a placebo-controlled phase 3 clinical trial in Duchenne muscular dystrophy (DMD). The drug, Givinostat, is a HDAC inhibitor, trialed with the aim of slowing disease progression through activating muscle repair mechanisms to increase muscle fibre regeneration, reduce inflammation and reduce fibrosis.

179 ambulant boys (aged 6-17) were dosed with either the drug or placebo over 19 months. The data, presented yesterday by Italfarmaco at the US Parent Project Muscular Dystrophy (PPMD) annual conference, shows a statistically significant drug effect in slowing down muscle degeneration and reducing muscle inflammation and fibrosis, measured by the stair climb test and other functional tests.

The trial recruited patients globally, including in the UK through Duchenne UK’s DMD Hub, and was delivered at four sites funded by Duchenne UK (Newcastle, Alder Hey, Oswestry and Great Ormond Street Hospital.)

Duchenne UK aims to work with Italfarmaco to support the next steps towards UK regulatory approvals to ensure that everyone living with Duchenne, who could benefit from this treatment, has access to it.

For further information, please read the press release here.

If your child has taken part in this trial and you have any questions, please contact us at [email protected].

Duchenne UK will be sharing the data at the next Parent Information Day on Saturday 1st October; further information will follow, and registration will open in the coming weeks.





Published on 26 June 2022

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