Orphan drug: Latest News, Top Stories & Analysis

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received orphan medicinal product designation from the European Medicines Agency (EMA) for OCU410ST, a promising treatment for ABCA4-associated retinopathies including Stargardt disease, …

Regional

Cabaletta Bio Receives FDA Orphan Drug Designation for Innovative Autoimmune Disease Therapy

March 24, 2024March 23, 2024 - by Timothy Alexander

PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to its investigational therapy, CABA-201. This designation …