This development marks the first nonsteroidal medication authorised to treat DMD patients with all genetic variants of a rare neurological disorder.
A histone deacetylase (HDAC) inhibitor, Duvyzat can lower inflammation and muscle loss by targeting pathogenic processes.
The approval is based on findings from the multicentre, randomised, double-blind, and placebo-controlled Phase III EPIDYS clinical trial in 179 ambulant boys.
It assessed Duvyzat given twice daily versus placebo, in addition to glucocorticosteroid treatment in both study arms.
The trial met the primary endpoint with subjects in the Duvyzat arm having a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. The mean change was 1.25 seconds for Duvyzat patients versus 3.03 seconds for placebo.
A secondary endpoint, the North Star Ambulatory Assessment (NSAA), also indicated less deterioration in physical function in Duvyzat-treated patients after 18 months.
Diarrhoea, abdominal pain, lowered platelets leading to increased bleeding, and nausea/vomiting among others were reported to be the common side effects linked to Duvyzat.
The FDA provided priority review and fast track designation for Duvyzat, which also received orphan drug and rare paediatric disease designations.
Italfarmaco Group chief medical officer Paolo Bettica said: “The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development programme, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD.
“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval. Our focus now is to make Duvyzat available as a treatment for DMD management in the US as quickly as possible.”