Regulation & Policy

The European Medicines Agency has given a positive opinion to a new immune-stimulating therapy for multiple myeloma using an accelerated procedure that is reserved for medicines addressing a major public health need.

The US Food and Drug Administration has approved the first drug to show a survival benefit in liposarcoma, a type of soft tissue sarcoma that is difficult to diagnose and treat. The drug, Halaven (eribulin mesylate), is a chemotherapy developed by Eisai Inc.

A volunteer in a Phase 1 trial of a novel neurological therapy, who had been declared brain dead three days ago, died on Sunday, according to the drug’s developer Bial-Portela & Ca. SA of Portugal. Five other patients who took part in the trial remained hospitalised.

A candidate drug for Duchenne muscular dystrophy, a genetic disorder that progressively weakens muscles in the body, has been turned down by the US Food and Drug Administration because of insufficient efficacy, according to the sponsor BioMarin Pharmaceutical Inc.

A product that is currently used to treat burns has now been approved in the US for certain diabetic foot ulcers. It is a matrix which consists of silicone, cow collagen and shark cartilage and is placed over the ulcer providing an environment for new skin and tissue to regenerate and heal the wound.

 A new lung cancer treatment for patients who have a specific tumour mutation has received a positive opinion in Europe. The drug, Tagrisso (osimertinib), was developed by AstraZeneca Plc.

It is not often that regulators, companies and investors meet in the same room, let alone discuss topics of mutual interest. But this was the setting for the fourth annual meeting of the industry group, European Biopharmaceutical Enterprises (EBE), and the European Medicines Agency (EMA) in London on 14 December. 

The US Food and Drug Administration (FDA) has rejected a new indication for Bristol-Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab) saying that more data is required. The proposed indication is for BRAF V600 positive metastatic melanoma.

The first monoclonal antibody for multiple myeloma has been approved by the US Food and Drug Administration under its accelerated approval procedure. Darzalex (daratumumab), was developed by Genmab A/S and licensed to Janssen Biotech Inc in 2012.

Guido Rasi has been appointed to a new five-year term as executive director of the European Medicines Agency following a fresh recruitment process. Prof Rasi had been forced to step down a year ago in the wake of a procedural challenge to his earlier appointment.