FDA denies Barth syndrome drug approval, suggests accelerated pathway
For the previous decade, Stealth BioTherapeutics has ridden a rollercoaster making an attempt to persuade the Meals and Drug Administration to approve its ultra-rare illness drug. Now, the corporate has encountered one more twist — an surprising regulatory rejection that won’t solely delay entry and pressure its funds, however guarantee a few of the most weak sufferers are denied the remedy.
At subject is a drugs for Barth syndrome, a uncommon sickness that causes an enlarged coronary heart, muscle weak spot and a shortened life expectancy. The illness afflicts as much as 150 individuals within the U.S., an especially small quantity that has, at occasions, made it tough for the corporate and the FDA to discover a approach to generate sufficient of the proper of research knowledge to make the drug accessible to sufferers.
However after bouncing between totally different company divisions and practically ending its improvement efforts, Stealth final October gained a key victory — a majority of panelists on an FDA advisory committee voted to suggest approval. The FDA, nonetheless, then missed a beforehand scheduled deadline this previous January to finish its advertising evaluate, pushing a call to April. However the company missed that deadline, too, suggesting the huge variety of job cuts had been an element.
This text is unique to STAT+ subscribers
Unlock this text — plus in-depth evaluation, newsletters, premium occasions, and information alerts.
Have already got an account? Log in
To learn the remainder of this story subscribe to STAT+.
