USA—The Food and Drug Administration has granted approval for a groundbreaking drug to treat Duchenne muscular dystrophy, marking a significant milestone in the medical landscape.

Duvyza, developed by Italian pharmaceutical company Italfarmaco, has will now be available for use in all Duchenne patients aged six and above, irrespective of their disease’s genetic profile.

The drug will be distributed in the U.S. by Italfarmaco’s subsidiary, ITF Therapeutics.

Duvyza represents a revolutionary advancement as the first non-steroidal treatment for Duchenne muscular dystrophy.

Administered in pill form, it is designed to mitigate inflammation and muscle degeneration. Clinical trials have demonstrated statistically significant improvements in motor function when compared to a placebo.

Operating as an HDAC inhibitor, Duvyza targets enzyme activity associated with muscle damage, thereby slowing the deterioration of muscle fibers characteristic of Duchenne muscular dystrophy.

When used in conjunction with steroids, the drug has shown promising results in enhancing patients’ ability to climb stairs and improving overall movement, as indicated by recent findings published in The Lancet.

While the pricing for Duvyza is yet to be determined, healthcare providers are required to assess patients’ platelet counts and triglyceride levels before prescribing the drug.

Monitoring of these levels is advised during treatment. Common side effects include diarrhea, stomach pain, nausea, and vomiting.

The approval of Duvyza signals a significant shift in the treatment landscape for Duchenne muscular dystrophy, a debilitating and often fatal neuromuscular disorder.

Since 2016, the FDA has greenlit four “exon skippers” and approved the first gene therapy, Elevidys, by Sarepta Therapeutics, offering hope for improved outcomes.

Santhera Pharmaceuticals’ Agamree, a newer steroid drug, was also cleared by the FDA in 2023.

However, existing treatments are tailored to specific subsets of patients, leaving many with limited options. Steroids, though commonly used, only offer symptomatic relief without addressing the root cause of the disease.

Duvyza’s approval now presents patients with the potential to enhance the efficacy of current treatments.

Debra Miller, founder and CEO of patient advocacy group CureDuchenne, lauded Duvyza’s approval as a significant stride towards accelerating transformative treatments for all patients, irrespective of their genetic mutations.

Patient advocates have expressed enthusiasm for Duvyza, viewing it as vital in advancing combination therapy and developing a universal treatment protocol for Duchenne patients.

ITF Therapeutics has affirmed its commitment to expeditiously making Duvyza available to eligible Duchenne patients in the U.S., underscoring the company’s dedication to addressing unmet medical needs.

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