Givinostat (Duvyzat), a histone deacetylase inhibitor, is the first nonsteroidal drug approved to treat patients aged 6 years or older with all genetic variants of DMD.
Givinostat, a histone deacetylase inhibitor, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It works by targeting pathogenic processes to reduce inflammation and loss of muscle, the FDA explained in"DMD denies the opportunity for a healthy life to the children it affects.
Although results in the four-stair climb assessment from baseline to 72 weeks, the trial's primary endpoint, worsened in both the treatment and placebo groups, the decline was significantly smaller with givinostat,"There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients," study investigator Craig M. McDonald, MD, with UC Davis Health, said in a company news release.
Givinostat works differently than conventional DMD treatments, suggesting "the combination of this new drug with other therapies may further improve the lives of our patients living with DMD," added MDA's chief medical officer Barry Byrne, MD, PhD.
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