The new Prescription Drug User Fee Act target date is April 29, 2025. The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for ...
Three months ago, the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in support of elamipretide becoming the first approved treatment for Barth syndrome, an X-linked genetic ...
If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.
The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.
Barth syndrome (BTHS) is a rare genetic disorder primarily affecting males, characterized by a range of symptoms including cardiomyopathy, skeletal myopathy, and neutropenia. The condition is ...
The table below is a review of notable updates that occurred in January 2025 for investigational products in development.
The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.
The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome. The ...