EU Grants Conditional Approval to Italfarmaco's Duvyzat for DMD Treatment

Italfarmaco's Duvyzat (givinostat) has received conditional approval from the European Commission to treat Duchenne muscular dystrophy (DMD), marking a significant advancement in medical treatment for this condition. This approval comes more than a year after the drug secured FDA authorization in the United States. Specifically designed as an oral HDAC inhibitor, Duvyzat is intended for ambulatory patients aged six and older who are also on corticosteroid therapy, with no restrictions pertaining to their genetic mutation.

The president of Italfarmaco, Francesco De Santis, expressed satisfaction with this development, highlighting that the approval provides a new treatment option for a broad spectrum of patients suffering from DMD. This decision by the European Commission was influenced by a recommendation from the European Medicines Agency's (EMA) drug advisory committee, which had reviewed the data earlier in April.

The approval was substantiated by findings from the Phase III EPIDYS study, which included 179 ambulatory boys diagnosed with DMD. Participants were randomized to receive either Duvyzat or a placebo, alongside the standard corticosteroid treatment. The study's outcomes demonstrated that Duvyzat met its primary endpoint: patients who received the drug experienced a significantly slower decline in their ability to climb four stairs compared to those who were given a placebo.

To further bolster the case for Duvyzat's full approval, Italfarmaco is conducting additional confirmatory studies. Alongside its approval in the EU and the US, Duvyzat has also been conditionally approved in the UK for ambulatory patients aged six and above, and for non-ambulatory patients.

The introduction of Duvyzat to the European market places it alongside Santhera Pharmaceuticals’ Agamree (vamorolone), another DMD treatment recently authorized in the EU. It is noteworthy that Sarepta Therapeutics faced setbacks with its own DMD therapies; the EMA rejected their exon-skipping drug Exondys 51 (eteplirsen) in 2018, and studies on their gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), were recently paused due to a patient death associated with liver failure.

Overall, the conditional approval of Duvyzat by the European Commission represents a promising advancement for individuals living with Duchenne muscular dystrophy, offering them new hope and treatment possibilities. Italfarmaco's commitment to conducting further studies underscores the importance of continuous research and validation in order to achieve full approval and maximize the drug's potential benefits for patients in the long term.