FDA approves NS Pharma’s Viltepso for Duchenne muscular dystrophy, but concerns over benefit persist

pharmafile | August 13, 2020 | News story | Sales and Marketing Duchenne Muscular Dystrophy, FDA, NS pharma, Viltepso 

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy.

Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin. This indication accounts for around 8% of DMD patients, and marks the first exon 53 skipping therapy in the US to increase dystrophin in children as young as four years old.

The drug was trialled in two studies of a total of 32 male participants with genetically confirmed DMD. In one of these studies of 16 patients, Viltepso generated an average increase in dystrophin levels from 0.6% of normal at baseline to 5.9% of normal 25 weeks after treatment in eight participants receiving the recommended dose.

Based on these data, the FDA judged that Viltepso is “reasonably likely” to provide clinical benefit in the approved indication. However, while the US regulator gave its authorisation to the drug, it has requested that NS pharma carry out an additional clinical study to confirm this benefit and ensure continued approval. If this benefit cannot be proven, the FDA may withdraw its authorisation.   

“For decades, neurologists who treat DMD have hoped for the discovery of therapies capable of significantly improving dystrophin production, and the magnitude of dystrophin increases observed with Viltepso are impressive,” said study investigator Dr Vamshi Rao of Ann & Robert H Lurie Children’s Hospital in Chicago. “The approval of Viltepso is an exciting development for DMD patients amenable to exon 53 skipping therapy and may rapidly become a foundational treatment for these patients.”

Matt Fellows

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