Italfarmaco’s Duvyzat gains EC conditional approval for DMD treatment

Duvyzat must be administered alongside corticosteroids and is not limited by the patient’s specific genetic mutation.

The decision is applicable across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway.

Following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in April 2025, this approval marks a significant milestone.

The EC’s authorisation is based on the Phase III EPIDYS study, where 179 ambulant boys aged six years or above were administered either Duvyzat or placebo alongside corticosteroids.

The study achieved its primary endpoint by showing improvement in time taken to complete the four-stair climb assessment with Duvyzat.

Additionally, patients treated with Duvyzat experienced a 40% slower decline in cumulative loss of north star ambulatory assessment (NSAA) items compared to those on placebo. This suggests that the treatment may help delay disease progression among individuals affected by DMD.

Italfarmaco Group chief medical officer Paolo Bettica said: “People living with DMD in Europe have long awaited new therapeutic options that can alter the course of this devastating disease. Until now, there have been limited approved treatments that address the underlying pathology of DMD across the broad patient population.

“That changes with the approval of Duvyzat, which slows disease progression and preserves muscle function — regardless of the gene mutation — by targeting disease mechanisms.”

While additional clinical studies are underway to further verify and detail its therapeutic benefits, the conditional marketing authorisation allows wider access to ambulant patients within Europe who could potentially benefit from this treatment option.

Approvals have also been secured with the US Food and Drug Administration (FDA) in March 2024 for similar patient demographics.

In addition, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved it for ambulatory patients aged six years or above while granting conditional approval for non-ambulatory individuals.