Europe Greenlights Duvyzat: A New Dawn in the Fight Against Duchenne's Devastation!
- European Commission grants conditional approval for Duvyzat (givinostat) to treat Duchenne Muscular Dystrophy (DMD).
- The novel drug offers hope by slowing disease progression for ambulant patients aged 6+, regardless of genetic mutation.
- Based on the pivotal EPIDYS trial, Duvyzat showed significant delay in muscle function decline.
In a landmark decision that reverberates with hope across Europe, the European Commission (EC) has granted conditional marketing authorisation for Duvyzat (givinostat), a pioneering treatment poised to change the relentless course of Duchenne muscular dystrophy (DMD). This watershed moment, announced by Italfarmaco, follows a positive CHMP opinion on April 25th, 2025, and unlocks a new therapeutic avenue for ambulant patients aged six and older in all 27 EU member states, plus Iceland, Liechtenstein, and Norway.
For too long, families have watched DMD, a cruel genetic thief, progressively steal muscle and mobility. But Duvyzat, a novel histone deacetylase (HDAC) inhibitor, offers a powerful counter-strike. Unlike many treatments, its innovative mechanism targets the overactive HDAC enzymes that fuel muscle inflammation and loss, working independently of the specific genetic mutation causing the disease1, 2, 3. This means a broader range of young warriors can now benefit from a therapy designed to preserve precious muscle strength and function when taken with corticosteroids.
The approval rides on the compelling results of the EPIDYS study, where Duvyzat significantly slowed disease progression. Boys treated with Duvyzat demonstrated a clinically meaningful difference in completing a four-stair climb and showed a remarkable 40% less decline in the North Star Ambulatory Assessment. Crucially, long-term data suggests Duvyzat could delay the loss of ambulation by nearly three years, pushing the median age to 18.1 years compared to 15.2 years in controls.
This European milestone, hot on the heels of FDA approval in March 20243 and UK MHRA authorisation, ignites renewed optimism. Italfarmaco's commitment extends beyond DMD, with Duvyzat's multi-targeted approach also being explored for Becker muscular dystrophy4, 5, underscoring a relentless pursuit of therapies for rare diseases. As Italfarmaco works to ensure swift access, the DMD community can finally embrace a treatment that truly targets the underlying pathology, offering not just hope, but a tangible weapon against this devastating condition6, 8.
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