For the previous decade, Stealth BioTherapeutics has ridden a rollercoaster attempting 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 among the most weak sufferers are denied the therapy.
At challenge is a medicine for Barth syndrome, a uncommon sickness that causes an enlarged coronary heart, muscle weak point and a shortened life expectancy. The illness afflicts as much as 150 folks within the U.S., an especially small quantity that has, at instances, made it troublesome for the corporate and the FDA to discover a technique to generate sufficient of the correct of research knowledge to make the drug accessible to sufferers.
However after bouncing between completely 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, nevertheless, then missed a beforehand scheduled deadline this previous January to finish its advertising overview, pushing a call to April. However the company missed that deadline, too, suggesting the huge variety of job cuts had been an element.
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