a first-in-class mitochondria-targeted therapeutic in development for individuals with Barth syndrome. The new PDUFA target action date is April 29, 2025. Stealth BioTherapeutics Logo (PRNewsFoto ...

The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.

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 ...

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.

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 ...

The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.

how to correct it", says Barth. "Neurogenerative diseases for example can be the result of misregulated genes during early stages of pregnancy. In fact, there are a couple of severe diseases, such as ...

Maharashtra has reported its first death due to Guillain-Barre Syndrome (GBS). A Pune-based Chartered Accountant was suffering from diarrhoea for a few days. Later, he could not move his hands and ...

A man in Maharashtra, who was suspected to have Guillain-Barre Syndrome (GBS) died in the Solapur district today while the number of cases of the immunological nerve disorder crossed 100 in Pune.

A Prescription Drug User Fee Act data of November 18, 2025 has been assigned to the application. The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ...