FDA says no to BioMarin’s Duchenne Muscular Dystrophy drug
pharmafile | January 14, 2016 | News story | Medical Communications, Sales and Marketing | BioMarin, Duchenne Muscular Dystrophy, FDA, Kyndrisa, drisapersen
The FDA has rejected BioMarin’s Duchenne Muscular Dystrophy (DMD) drug Kyndrisa, saying there was not enough evidence that the drug was effective in treating the rare muscle wasting condition.
The FDA also raised concerns about potential long-term side effects, including kidney damage and a reduction in the number of blood platelets.
BioMarin says the US regulator issued a Complete Response letter to its New Drug Application (NDA) for Kyndrisa (drisapersen), adding that it will work with the FDA to determine the appropriate next steps for the application.
Duchenne affects approximately one in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood. There is currently no FDA-approved therapy designed specifically to treat Duchenne.
The California-based company added that it would continue ongoing Kyndrisa extension studies and pointed out that it still awaits a decision from European regulators for the drug, expected in the first half of 2016.
While a disappointment to BioMarin, the FDA’s rejection of Kyndrisa could be considered good news for PTC Therapeutics, which is also working on a DMD treatment. The New Jersey-based company’s drug, Translarna (ataluren) was initially rejected by UK watchdog NICE in draft guidance, but awaits a final decision, and could be approved if PTC is able to provide sufficient evidence of its safety and efficacy.
The drug failed to meet its primary endpoint in a Phase III trial, as revealed in data published in October, but PTC believes it showed enough benefit for the FDA to consider an approval, particularly as there is a significant unmet need in treatment options for the rare disease.
The FDA will also review another DMD drug from Sarepta Therapeutics, eteplirsen, next week, a treatment which faces a similarly limited efficacy profile with currently-available data.
Joel Levy
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