Orphan Drug Designation - United States News Beep

U.S. FDA Accepts Teva’s New Drug Application (NDA) and Grants Priority Review for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Patients with Tourette Syndrome

August 20, 2026

Teva Pharmaceutical Industries Ltd Ecopipam (EBS-101) is a first-in-class selective D1 (dopamine) receptor antagonist with Orphan Drug designation NDA…

MMedication

Praxis Precision Medicines Announces FDA Acceptance and Priority Review of New Drug Application for Relutrigine in Patients with SCN2A and SCN8A DEEs

March 31, 2026

Praxis Precision Medicines, Inc. FDA assigned PDUFA target action date of September 27, 2026 BOSTON, March 30, 2026…

GGenetics

Servier acquires potential treatment for Fragile X syndrome, the most common genetic cause of autism spectrum disorder

September 8, 2025

Servier acquires KER-0193, a potential treatment for Fragile X syndrome, developed by Kaerus Bioscience, a Medicxi company KER-0193,…