Regulation & Policy

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.

Novo Nordisk A/S has become the latest producer of a diabetes medicine to stop supplying the German market following a disagreement over pricing. Novo will cease supplying Tresiba (insulin degludec) in Germany after 30 September 2015.

The European Medicines Agency has recommended the approval of the first histone deacetylase (HDAC) inhibitor for the treatment of multiple myeloma, a rare cancer of the white blood cells. The drug, Farydak (panobinostat), was developed by Novartis.

A new implantable deep brain stimulation device has been approved by the Food and Drug Administration for patients with symptoms of both Parkinson’s disease and essential tremor, two discrete disorders for which there are no medical cures.

Acting on evidence that trans-unsaturated fatty acids are a risk for heart disease, the Food and Drug Administration has ordered manufacturers of processed foods to reformulate their products to remove these ingredients, also known as trans fats.

The US Food and Drug Administration has approved sirolimus for the treatment of a rare, progressive lung disease after a clinical trial in 89 patients showed the drug was effective compared with a placebo. Sirolimus trades as Rapamune and is an approved immunosuppressive agent. 

Small and medium-sized enterprises (SMEs) play a special role in drug development particularly in the field of advanced therapy medicinal products (ATMPs). With small numbers of staff, many of whom are scientists, they are in a unique position to drive medical innovation.

The Brussels-based Innovative Medicines Initiative is launching three new projects around Alzheimer’s disease that will review current nomenclature, create a new network of patient data and test novel treatments in patients with early-stage disease.