FDA approves Italfarmaco’s drug Duvyzat for Duchenne muscular dystrophy

The FDA approved the first nonsteroidal drug to treat all genetic variants of Duchenne muscular dystrophy, clearing drugmaker Italfarmaco’s oral treatment for patients age 6 and older.

The drug, givinostat, will be sold as Duvyzat, the FDA said in a press release announcing the approval. It’s different than genetic medicines approved for Duchenne, such as exon-skipping drugs or gene therapy, which require patients to have certain mutations.

Duvyzat is a histone deacetylase inhibitor, taken twice a day orally. “It is not trying to restore the dystrophin, but it’s trying to counteract the downstream effect of the lack of dystrophin,” Paolo Bettica, Italfarmaco’s VP of R&D, told Endpoints News ahead of the decision. The approval follows a three-month delay by the FDA in December.

In a Phase 3 clinical trial of 179 boys with Duchenne who were walking and at least 6 years old, those who received Duvyzat performed better on a timed four-stair climb test than those who received placebo, according to a study published Tuesday in Lancet Neurology. The most common adverse events in the treatment group were diarrhea and vomiting.

Duchenne treatments have evolved substantially in the last decade, with accelerated approvals of four exon-skipping drugs for patients with certain mutations. The FDA last year granted a landmark accelerated approval to Sarepta’s gene therapy Elevidys for boys who are 4 and 5 years of age. And the agency is expected to decide by June whether to expand Elevidys’ label to a wider age range and whether to convert it to a full approval.

Thursday’s approval of Italfarmaco’s drug also could open the door to combination approaches for treating Duchenne.

“Given the mechanism of action and given what is happening, this is something that potentially in the future we might be considering and studying,” Bettica said.

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