hotspot

Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug, Viltepso, part of a controversial class of treatments for the rare disease, had failed to reach its primary endpoint in a placebo-controlled confirmatory trial.

Children who received the drug could stand up faster from the floor at the end of the study, but so could children who received a placebo. There was no statistically significant difference between the groups.

advertisement

Although Nippon did not release detailed data, the failure is notable because Viltepso belongs to a contentious class of drugs called exon-skippers. Duchenne is caused by mutations in a muscle-supporting gene called dystrophin. These drugs are designed to increase the amount of dystrophin protein that patients with specific mutations produce. But the drugs only lead to slight increases in protein levels, and their clinical benefits have never been proven in a large, placebo-controlled study.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Already have an account? Log in

Monthly

$39

Totals $468 per year

Totals $468 per year

Starter

$30

for 3 months, then $39/month

Then $39/month

Annual

$399

Save 15%

Save 15%

11+ Users

Custom

Savings start at 25%!

Savings start at 25%!

2-10 Users

$300

Annually per user

$300 Annually per user

Get unlimited access to award-winning journalism and exclusive events.