Italfarmaco has secured conditional marketing authorisation from the European Commission (EC) for Duvyzat for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients aged six and older.
This approval applies to patients regardless of the genetic mutation involved, provided Duvyzat is administered alongside corticosteroids. Duvyzat is a histone deacetylase inhibitor.
The European Medicines Agency’s Committee for Medicinal Products for Human Use had previously endorsed this decision in April 2025. This authorisation extends to all 27 European Union (EU) member states, as well as Iceland, Liechtenstein, and Norway.
Italfarmaco said that it is now collaborating with national authorities to ensure Duvyzat is accessible throughout Europe.
Italfarmaco 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.”
The approval follows positive outcomes from the EPIDYS Phase 3 trial, which involved 179 ambulant boys over the age of six receiving either Duvyzat or a placebo in conjunction with corticosteroid therapy.
Results demonstrated a statistically significant improvement in the time required to complete a four-stair climb. Secondary endpoints also showed positive results, including the North Star Ambulatory Assessment and fat infiltration measured by MRI.
The treatment resulted in a 40% slower decline in cumulative loss of NSAA items, indicating potential for delaying disease progression. Most adverse effects were mild to moderate.
Findings were published in The Lancet Neurology in March 2024. Long-term data from an ongoing EPIDYS extension study showed that median age at loss of ambulation was 18.1 years with Duvyzat versus 15.2 years in control groups.
While conditional marketing authorisation has been granted in the EU, Italfarmaco will continue conducting clinical studies to confirm Duvyzat’s therapeutic benefits further.
In addition to European approval, Duvyzat was approved by the US Food and Drug Administration (FDA) in March 2024 for DMD patients aged six and above.
In the UK, it received approval from the Medicines and Healthcare products Regulatory Agency for ambulatory patients, with conditional marketing authorisation extended to non-ambulatory patients.
Duvyzat emerged from Italfarmaco’s research efforts alongside Telethon and Duchenne Parent Project Italy.
As an orally administered histone deacetylase inhibitor, it addresses excessive HDAC activity seen in DMD muscles, restoring essential gene expression and biological processes required for muscle maintenance and repair without depending on specific dystrophin gene mutations.
