EMA Approved a Record Number of Orphan Drugs Last Year

January 15, 2015

The European Medicines Agency approved a record number of orphan drugs in 2014, a milestone it says is likely due to increasing early collaboration with sponsors.

Out of the 82 drugs approved last year, 17 were for rare diseases, the EMA said, a full 21 percent of the approvals. Several of the orphan drugs were the first therapies to treat their respective diseases, including PTC Therapeutics’ Translarna (ataluren) for Duchenne muscular dystrophy and Clinuvel Pharmaceuticals’ Scenesse (afamelanotide) for erythropoietic protoporphyria.

Seventy percent of new drug sponsors took advantage of an EMA program to work more closely with the Committee for Medicinal Products for Human Use, the agency said, up from 50 percent in 2013.

Orphan drugs approved last year included eight new cancer medicines, including AstraZeneca’s Lynparza (olaparib), Janssen’s Imbruvica (ibrutinib), Roche’s Gazyvaro (obinutuzumab), GlaxoSmithKline’s Mekinist (trametinib) and Eli Lilly’s Cyramza (ramucirumab).

Other orphan drug products included Boehringer Ingelheim’s Ofev (nintedanib) for idiopathic pulmonary fibrosis, Janssen’s Sylvant (siltuximab) for multicentric Castleman’s disease, and Laboratoire HRA Pharma’s Ketoconazole HRA for Endogenous Cushing’s Syndrome.

To qualify as an EU orphan drug, the product must treat a disease that is life-threatening or chronically debilitating and affects no more than five in every 10,000 people. — Lena Freund