Regulation & Policy

For more than a year, the European Medicines Agency (EMA) has been meeting individually with a number of companies that are developing products for difficult-to-treat diseases to speed the path towards registration. The discussions are non-binding, which means that regulatory approval is not assured. But they give both sides a chance to discuss the requirements of a good clinical programme, including the sort of information that will eventually be needed to show effectiveness in clinical practice.

Guido Rasi, a former head of the Italian Medicines Agency, has been nominated to lead the European Medicines Agency once again after a procedural challenge to his appointment in 2011 forced him to step down in November 2014.

The European Medicines Agency has given a positive opinion for a new Amgen Inc medicine that has shown an ability to kill or slow down the growth of multiple myeloma cells by blocking the cells’ proteasome.

The US Food and Drug Administration has extended the approved indication for the blood-thinning drug Brilinta (ticagrelor) to include the long-term use in patients with a history of heart failure. The approval was announced on 3 September.

Repatha (evolocumab), a new drug for lowering cholesterol in patients with cardiovascular disease or familial hypercholesterolemia, has been approved by the Food and Drug Administration. Developed by Amgen, the drug received a positive opinion from the European Medicines Agency in May.

A new drug intended to treat low sexual desire in women has been approved by the US Food and Drug Administration. Addyi (flibanserin) is the first pharmaceutical for this condition, which is known as hypoactive sexual desire disorder (HSDD).

 A new vaccine designed to protect young children in sub-Saharan Africa from infection from thePlasmodium falciparum parasite has been given a positive opinion by the European Medicines Agency even as its effectiveness was described as modest.

Proposed legislation that would increase funding for US biomedical research and promote personalised medicine passed the House of Representatives on 10 July and was sent to the Senate for debate.

Following a request from Denmark, the European Medicines Agency has begun a review of the safety of the human papillomavirus vaccines Gardasil and Cervarix to find out whether or not reports of two rare adverse events are linked to the vaccines.

Vertex Pharmaceuticals Inc has won approval in the US for a second drug to treat cystic fibrosis, this time for a treatment targeting the F508del mutation, the most common cause of the disease. Called Orkambi (lumacaftor/ivacaftor), the drug is indicated for patients who have two copies of the mutation.