Regulation & Policy

Business hubs where life science companies co-locate in order to share infrastructure exist almost everywhere. But not all business hubs support basic science, let alone national healthcare systems. EIT Health is different. The group is part of the European Institute of Innovation and Technology which was set up in 2015 to promote the interface between industry, science and public healthcare systems. At conference in Paris on 2 to 3 December, members of the consortium met to discuss issues facing healthcare – and to congratulate a new crop of entrepreneurs.

The US Food and Drug Administration has approved a new drug for Duchenne muscular dystrophy (DMD) on the basis of data showing that the medicine increased the production of  the dystrophin protein in patients – a surrogate for improved motor function. The drug, Vyondys 53, was developed by Sarepta Therapeutics Inc, whose first DMD drug Exondys 51 was approved by the FDA in 2016, also on the basis of a surrogate endpoint.

The first vaccine for the active immunisation of individuals at risk of infection from the Ebola virus has been cleared by the World Health Organization for procurement by the United Nations and included in a stockpile for use in countries affected by the disease. These countries include the Democratic Republic of Congo where a public health emergency has been declared by the WHO.

A new drug for anaemia in patients with beta thalassaemia, a rare blood disorder, has been approved for marketing in the US following clinical testing which showed that patients receiving the treatment needed fewer blood transfusions than those on a placebo. The drug, Reblozyl (luspatercept-aamt), was authorised by the Food and Drug Administration on 8 November.

The US Food and Drug Administration has approved a new diagnostic capable of detecting antiretroviral drug resistance mutations in people living with the human immunodeficiency virus (HIV) Type 1. The test is able to detect mutations from a simple blood test that uses next generation sequencing to identify the genetic alterations.

The safety committee of the European Medicines Agency is recommending that new restrictions be placed on the use of the multiple sclerosis drug Lemtrada (alemtuzumab) in light of reports of rare but serious side effects, including deaths. The latest recommendations replace temporary measures issued in April. They will be considered by the agency’s main scientific committee for a formal opinion.

A second experimental Ebola vaccine is to be tested in a large clinical trial in the Democratic Republic of Congo (DRC) as public health authorities mobilise resources to contain an outbreak of Ebola virus disease, declared by the World Health Organization to be a public health emergency.

Kiadis Pharma NV has strengthened its scientific advisory board to guide the ongoing development of its cell therapy for patients who’ve had a stem cell transplant. This follows guidance from the European Medicines Agency that the product, ATIR101, will not be recommended for approval. ATIR101 has been under regulatory review since 2017.

Biogen Inc and Eisai Co Ltd have announced plans to seek US regulatory approval for aducanumab, a candidate treatment for early Alzheimer’s disease, just months after two Phase 3 trials for the drug were stopped for futility. The decision to proceed with registration follows discussions with the Food and Drug Administration about a larger dataset from the Phase 3 studies which showed that the drug worked at a high dose.

A new triple combination therapy for cystic fibrosis, targeting the most common genetic mutation behind the disease, has been approved by the US Food and Drug Administration. Trikafta (elexacaftor/ivacaftor/tezacaftor) is now available to patients 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is said to represent about 90% of all patients with the disease.