Regulation & Policy

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.

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.

Merck & Co Inc, whose checkpoint antibody Keytruda (pembrolizumab) already has 20 approved indications, received a further endorsement on 18 June with US Food and Drug Administration approval for the treatment of metastatic small cell lung cancer. Keytruda is already approved as a first-line treatment for non-small cell lung cancer. The new indication means the antibody can be used as a monotherapy for patients with small cell lung cancer whose disease has progressed on or after platinum-based chemotherapy and at least one other prior line of therapy.

Novo Nordisk A/S has broadened its coverage of the market for diabetes treatments following  the US approval on 17 June of a new indication for Victoza (liraglutide), its glucagon-like peptide-1 (GLP-1) receptor agonist. The new indication is for the treatment of paediatric patients 10 years or older with Type 2 diabetes. Victoza is the first non-insulin drug approved for the treatment of Type 2 diabetes in children since metformin was authorised for paediatric use in 2000. Victoza has been approved to treat adult patients with the disease since 2010.

The first diagnostic test for Zika virus infection, which can lead to birth defects if contracted during pregnancy, was approved on 23 May by the US Food and Drug Administration. The test, developed by InBios International Inc, is designed to detect antibodies to the pathogen which indicate that the body has generated an early immune response. It is meant to be used for people who show clinical signs of the disease or who have lived or travelled to Zika-infected areas. Prior to this decision, the InBios’ test and others, were authorised for emergency use only.

A new gene therapy has been approved in Europe, paving the way for its use in patients with beta-thalassaemia, a rare blood disorder. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March and a conditional approval by the European Commission on 29 May.

The US Food and Drug Administration has approved an antibody-drug conjugate from Roche for diffuse large B cell lymphoma (DLBCL), an aggressive cancer that grows quickly in the lymph nodes and may affect the bone marrow, spleen, liver or other organs. The drug targets CD79b, a protein expressed on B cells.