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
$39/month Get StartedTotals $468 per year
Starter
$30
for 3 months, then $39/month
$30 for 3 months Get StartedThen $39/month
Annual
$399
Save 15%
$399/year Get StartedSave 15%
11+ Users
Custom
Savings start at 25%!
Request A Quote Request A QuoteSavings start at 25%!
2-10 Users
$300
Annually per user
$300/year Get Started$300 Annually per user
View All Plans
Get unlimited access to award-winning journalism and exclusive events.
Subscribe Log In
Previous article：
ASCO: AstraZeneca says CAR
Next article： Jerome Adams on the need for new antivirals for Covid
Next article： Jerome Adams on the need for new antivirals for Covid
