Regulation & Policy

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.

Novartis has decided to price its new gene therapy for spinal muscular atrophy using a formula that measures the performance of the drug over five years. The therapy, Zolgensma (onasemnogene abeparvovec-xioi), is a one-time treatment for a genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.

The US Food and Drug Administration has approved a gene therapy to treat children under the age of two years who have spinal muscular atrophy, a rare genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.

Italian-born Mauro Ferrari, who studied medicine in the US and advised the US National Cancer Institute, is to be the next president of the European Research Council, an EU body that funds investigator-driven scientific research. Professor Ferrari will take up his position on 1 January 2020. He is known within the science community for his expertise in leveraging nanotechnology to treat and diagnose cancer.

Roche has secured a new indication for its antibody-drug conjugate Kadcyla (trastuzumab emtansine) that extends the drug’s approved use to early breast cancer in patients who have had surgery but still show residual signs of disease. Treatment would take place after chemotherapy and antibody therapy. Kadcyla was first approved for late-stage breast cancer in 2013. In both cases, patients have tumours that overexpress the HER2 protein.

The US Food and Drug Administration has issued its first-ever draft guidance on the development of stimulant drugs to treat attention deficit hyperactivity disorder (ADHD), a condition whose symptoms include inattention and high levels of activity. While many of the recommendations are already in practice, the document gives clarification on the agency’s expectations for clinical trial design.