Six Drugs Snag Orphan Drug Designation in EU

Six new drugs snagged orphan drug designations from the European Medicines Agency’s Committee for Orphan Medicinal Products.

BioMarin’s hemophilia drug BMN270 was one of the recipients, as was Escala Therapeutics’ ManNAc for the treatment of GNE myopathy, Diamond BioPharm’s fosbretabulin tromethamine for the treatment of gastro-entero-pancreatic neuroendocrine tumors and Pharma Gateway’s DTX301 for the treatment of ornithine transcarbamylase deficiency.

Acerta Pharma’s acalabrutinib landed two orphan drug designations for different indications: one for the treatment of lymphoplasmacytic lymphoma and the other for mantle cell lymphoma.

Pharm Research Associates’ NY-ESO-1 esophageal squamous cell carcinoma-1 protein also nabbed an orphan status for the treatment of soft tissue sarcoma.