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contemporarypediatrics15d
Elamipretide to treat Barth syndrome sees FDA target action delay
This week, Stealth BioTherapeutics Inc. announced that the FDA did not meet the April 29, 2025, Prescription Drug User Fee Action (PDUFA) date for elamipretide's new drug application (NDA) to treat ...
The Pharma Letter15d
FDA misses decision deadline on Stealth’s Barth syndrome drug
US biotech firm Stealth BioTherapeutics (OTCQB: MITO) is facing renewed uncertainty after the American medicines regulator failed to deliver a decision on the company’s new drug application for ...
Yahoo15d
FDA misses approval deadline for biotech’s rare disease drug
The exterior of the Food and Drug Administration headquarters is seen on July 20, 2020 in White Oak, Maryland. 35,180 people played the daily Crossword recently. Can you solve it faster than ...
Mena FN15d
Stealth Biotherapeutics Announces Delay In FDA Action Date For Barth Syndrome Application
Barth syndrome is an ultra-rare, life threatening pediatric mitochondrial disease which is known to affect less than 150 individuals in the United States and less than 300 individuals worldwide.
Le Lézard16d
Barth Syndrome Foundation Calls for Urgent FDA Action Following Elamipretide Delay
The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments, and finding a cure for Barth syndrome ...
Yahoo Finance16d
Barth Syndrome Foundation Calls for Urgent FDA Action Following Elamipretide Delay
BOSTON, April 29, 2025--(BUSINESS WIRE)--The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments ...
Monthly Prescribing Reference16d
FDA Decision on Barth Syndrome Therapy Delayed Again
No revised PDUFA target date has been communicated by the FDA. A decision on the New Drug Application for elamipretide for the treatment of Barth syndrome has been delayed again, according to ...