The Food and Drug Administration approved the first nonsteroidal treatment for treating Duchenne muscular dystrophy (DMD) on Thursday evening.

The agency greenlit Duvyzat (givinostat) from Italfarmaco S.p.A., a Milan-based pharma company, to treat DMD, a severe neuromuscular disease that leads to progressive muscle degeneration and currently has no cure.

The drug is a histone deacetylase inhibitor that targets pathogenic processes to reduce inflammation and loss of muscle. As an oral medication, it’s administered twice daily and has common side effects like diarrhea, abdominal pain, a decrease in platelets, nausea/vomiting, an increase in triglycerides and a fever.

Duvyzat was approved to treat patients over the age of six based on the results of a Phase 3 study that found the drug met its primary endpoints of using a four stair climb to measure muscle function.

The approval came nine months after the FDA greenlit Sarepta Therapeutics’ Elevidys under the accelerated approval pathway, the first gene therapy to treat DMD.

“DMD denies the opportunity for a healthy life to the children it affects. The FDA is committed to advancing the development of new therapies for DMD,” said Emily Freilich, MD, director of the Division of Neurology 1, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a statement. “This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation.”

Prior to the full approval, the FDA had granted a priority review and fast track designation to Duvyet in addition to designations for orphan drug and rare pediatric disease.

Following the approval, Paolo Bettica, MD, PhD, who is the chief medical officer at Italfarmaco Group, commended the agency’s decision to approve a nonsteroidal option that can treat patients with all genetic variants of DMD.

“The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “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 U.S. as quickly as possible.”

The FDA’s action was also applauded by the Muscular Dystrophy Association (MDA), which called it “another significant treatment option” for people living with DMD. 

“As more drugs are approved for DMD we look forward to understanding if and how they might be used in combination,” Sharon Hesterlee, PhD, chief research officer at the MDA, said in a statement.

The approval builds on momentum in the DMD treatment space.

Months after Elevidys was approved, Sarepta said it failed to reach its primary endpoint in a Phase 3 trial. The single-dose gene transfer therapy didn’t reach statistical significance in the total assessment score to improve motor function, compared to placebo, in the study.

While that was a stepback, Dyne Therapeutics announced a few months later that its experimental treatment for DMD showed promise in data from a small Phase 1/2 trial.