Accelerated Viltepso approval at risk as confirmatory trial flops

28 May 2024

Japanese drugmaker Nippon Shinyaku's (TYO: 4516) US subsidiary NS Pharma has published a disappointing initial analysis from an important trial of Viltepso (viltolarsen).

A synthetic piece of nucleic acid known as an antisense oligonucleotide, Viltepso works by targeting a specific mutation in the Duchenne muscular dystrophy (DMD) gene.

The drug received Accelerated Approval in DMD from the US regulator in 2020, after trials showed it could increase dystrophin production.

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