EMA recommended approval for 40 new drugs in 2014
Asher Mullard
The European Medicines Agency (EMA) approved 40 new agents in 2014, up from 34 in 2013. This left the European regulators only one approval behind the US Food and Drug Administration (FDA) last year (see Nature Rev. Drug Discov. 14,77–81; 2015 for detailed analysis of the FDA's approvals in 2014). Both regulatory bodies set records with orphan-drug approvals, with each giving the green light to 17 agents for rare diseases.
Although there was considerable overlap between the EMA- and FDA-approval lists, the European regulators did act on a few agents before their US equivalents. The EMA is billing Chiesi Farmaceutici's Holoclar, an autologous human corneal epithelial cell product that includes stem cells for the treatment of limbal stem-cell deficiency, as the first stem-cell therapy to be approved in the European Union. The EMA also approved PTC Therapeutics' nonsense-mutation read-though small molecule ataluren for the treatment of muscular dystrophy, following a previous rejection and appeal from the drug company (Nature Biotech. 32, 706; 2014). The EMA also approved Clinuvel's afamelanotide, a synthetic peptide analogue of α-melanocyte-stimulating hormone, a potentially life-changing drug for patients with erythropoietic protoporphyria (Nature Biotech. 33, 7; 2015).