Regulation & Policy

The US Food and Drug Administration has approved a third cancer drug that targets a genetic driver of cancer, rather than a specific type of tumour. The drug, Rozlytrek (entrectinib), is directed at cancers with neurotrophic tyrosine receptor kinase (NTRK) gene fusions, a fault that can result in the activation of signalling pathways involved in the proliferation of a number of solid tumours. These fusions are known to occur in breast, colorectal, non-small cell lung, pancreatic and thyroid cancers, among others.

A new combination treatment for drug-resistant tuberculosis (TB) has been approved in the US and is under review at the European Medicines Agency. Developed by the Global Alliance for TB Drug Development Inc, it is only the third new drug approved for TB in more than 40 years, and the first to be produced by a not-for profit organisation.

Novartis has appointed Page Bouchard, an industry veteran, to the role of chief scientific officer at AveXis Inc, a subsidiary at the center of a controversy over data manipulation in relation to the recently approved gene therapy Zolgensma for spinal muscular atrophy (SMA). Dr Bouchard has been with Novartis for 10 years and will take over the roles of CSO and head of research and development at AveXis that were previously held by Brian Kaspar and Allan Kaspar.

Novartis has responded to a statement from the US Food and Drug Administration on 6 August in which the regulator raised the issue of manipulation of product testing data in the production process for Zolgensma, the recently approved gene therapy for spinal muscular atrophy.

The World Health Organization has added several cancer therapies to its Essential Medicines List in recognition of their contribution to extending the lives of patients with cancer. The list, which is updated every two years, is intended to guide health authorities on which medicines represent the best value for money based on their effectiveness.

Slovakia has been added to the group of European countries whose pharma manufacturing inspections systems are on par with those in the US, enabling the Food and Drug Administration to automatically recognise products produced in the country’s factories.

As a result, all 28 EU member states can now participate in a Mutual Recognition Agreement on good manufacturing practice (GMP) that was initiated in 1998, and slowly brought into force over the past 20 years. The European Commission announced the coming into effect of the full agreement on 11 July.

A new medicine for multiple myeloma has been approved by the US Food and Drug Administration giving patients whose disease is resistant to several other drugs, an option for treatment. The small molecule drug, Xpovio (selinexor), can now be used by patients who have received at least four other therapies, but without success. Resistance in these patients has been shown to proteasome inhibitors, immunomodulatory agents and an anti-CD38 monoclonal antibody.

Scott Gottlieb, the former Commissioner of the US Food and Drug Administration, has been elected to the board of Pfizer Inc where he will also serve on the board’s regulatory and compliance and science and technology committees.

Children 10 years and older will be eligible for treatment with Victoza (liraglutide), a non-insulin medication for Type 2 diabetes, assuming a positive opinion by the European Medicines Agency is cleared by the European Commission. The positive opinion was issued on 28 June, extending Victoza’s indication to children and adolescents with Type 2 diabetes. It is already approved for adults. Victoza is a glucagon-like peptide-1 (GLP-1) receptor agonist that slows digestion, prevents the liver from making too much glucose and helps the pancreas produce more insulin when needed.

A combination treatment for cystic fibrosis that targets patients with a specific genetic mutation, has now been approved in the US for children six years and older. The treatment, Symdeko (tezacaftor/ivacaftor), targets a common mutation in the cystic fibrosis gene – the F508del mutation – which can lead to severe respiratory and digestive problems. The cystic fibrosis gene encodes the cystic fibrosis conductance transmembrane regulator (CFTR) protein which controls the movement of chloride and sodium in and out of cells in the human body.