Within the newest twist over the destiny of an ultra-rare illness drug, Stealth BioTherapeutics mentioned it obtained “conflicting” indicators from the U.S. Meals and Drug Administration about info wanted to approve its remedy and, consequently, is making contingency plans to shut the corporate.
At subject is a remedy 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 folks within the U.S., a particularly small quantity that has, at instances, made it troublesome for Stealth and the FDA to agree on methods to generate sufficient of the correct of research information to make the drug obtainable to sufferers.
For the previous few years, the corporate has traveled an advanced path because the company bounced its advertising and marketing software between completely different divisions. Profitable FDA approval isn’t straightforward, however the sequence of occasions was generally cited by affected person advocacy teams for example of the necessity to persuade the company that approvals for uncommon illness medicine require extra flexibility.
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