2016 EMA drug approval recommendations

The European Medicines Agency (EMA) recommended approval for 27 new therapeutic agents in 2016, down from 39 in 2015 and 40 in 2014.

The EMA's count includes small molecules, antibodies, biologics, blood products, cellular therapies and vaccines, and so cannot be compared directly with approvals from the FDA's Center for Drug Evaluation and Research (CDER), which reviews only small molecules and some types of biologic. However, CDER also approved fewer products in 2016 than in previous years (see Nat. Rev. Drug Discov. 16, 73–76; 2017).

Most of the new EMA green lights went to therapeutics that the FDA approved in 2015 or 2016. There were a few notable differences, including approvals for two advanced cellular therapies that have not yet been approved by the FDA.

GlaxoSmithKline's strimvelis is the second gene therapy to be approved in Europe (Nat. Rev. Drug Discov. 15, 299; 2016). Strimvelis is an autologous gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). It is produced by collecting a patient's bone marrow cells and transducing these cells ex vivo with a viral vector so that they express adenosine deaminase (ADA).

The EMA also approved MolMed's zalmoxis, which consists of allogeneic T cells that have been genetically modified to carry a truncated form of the human low-affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2). The EMA approved the cellular product for haematopoietic stem cell transplantation, to aid immune reconstitution. HSV-TK Mut2 acts as a suicide switch, making the engineered T cells susceptible to ganciclovir or valganciclovir in case they induce graft-versus-host disease.

The EMA gave a first thumbs up to Eli Lilly's Janus kinase (JAK) inhibitor baricitinib for rheumatoid arthritis. The FDA recently pushed the Prescription Drug User Fee Act (PDUFA) date for this drug back to April 2017.

The EMA recommended two products that the FDA rejected in 2016: Amicus's migalastat, a pharmacological chaperone for Fabry disease, and Amgen's etelcalcetide, a calcimimetic for secondary hyperparathyroidism in adult patients with chronic kidney disease.