Even more orphan drugs!

In 2014, 41 new therapies received the green light from the FDA. This made 2014 the industry’s most prolific year since 1996, when 53 new drugs were authorised. Of these newcomers, 17 are designed for the treatment of rare conditions, pathologies affecting fewer than 200,000 Americans; this is the highest number of such drugs ever registered. Furthermore, almost two-thirds of these new drugs have not yet gone on sale overseas. Some of these authorisations benefited from accelerated assessment procedures including the “Priority Review Tag” and “Fast Track Review Route”. In Europe, the EMA approved a substantial number of orphan drugs, with 17 of the 82 new products approved this year focusing on conditions for which existing treatments are very limited or non-existent.