Regulation & Policy

The European Medicines Agency has issued new guidance to industry on Brexit, warning that all products under review by the UK regulatory authority will need to be transferred to rapporteurs or co-rapporteurs from the other EU member states. The rapporteur is the leader of the scientific team that evaluates applications from companies for marketing authorisations. The activities of this team are supported by the EMA secretariat. Once outside the EU, the UK’s Medicines and Healthcare products Regulatory Agency will no longer be eligible to participate in these evaluations.

The US Food and Drug Administration approved Libtayo (cemiplimab-rwlc) on 28 September for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) who are not eligible for curative surgery or radiation. The approval is the FDA’s first authorisation of a drug specifically for an advanced form of this disease.

The US Food and Drug Administration has approved Lumoxiti (moxetumomab pasudotox-tdfk), a new drug developed by AstraZeneca Plc for the treatment of refractory hairy cell leukaemia (HCL), a rare, slow-growing cancer of the blood.

The US Food and Drug Administration has issued a ‘complete response letter’ to GlaxoSmithKline Plc rejecting the company’s application for a third indication for mepolizumab (Nucala). The application was to use the drug as an add-on treatment for patients with chronic obstructive pulmonary disease (COPD).

Announcing the rejection on 7 September, GSK said the FDA is requiring more data to support the proposed indication.

Two US institutions have taken steps to simplify the regulation of gene therapy, and bring the oversight of these technologies into the existing framework for monitoring experimental medicines. The shift in policy is described by Francis Collins, director of the National Institutes of Health, and Scott Gottlieb, commissioner of the Food and Drug Administration, in an article in the New England Journal of Medicine on 15 August 2018.

China-based Zhejiang Tianyu Pharmaceutical Co Ltd has had its regulatory clearance for the supply of the active ingredient valsartan to the European market suspended following an inspection showing the presence of N-nitrosodimethylamine (NDMA), a suspected carcinogen. NDMA is classified as a probable human cardinogen based on the results of laboratory tests.

Oncology drugs that promise to meet a major public health need may in future be eligible for a clinical development pathway that is both simpler and less costly than traditional methods, the US Food and Drug Administration announced on 10 August.

The US Food and Drug Administration has approved an RNA therapeutic developed by Alnylam Pharmaceuticals Inc to treat patients with a rare genetic disease characterised by the build-up of abnormal amyloid protein in peripheral nerves and other organs.

A candidate treatment for Huntington’s disease has been selected by the European Medicines Agency for PRIME, a programme of regulatory support for developers of medicines that meet a major public health need. The drug, RG6042, is an antisense oligonucleotide.

It is being developed jointly by Roche and Ionis Pharmaceuticals Inc to slow or stop the progress of Huntington’s disease, a rare genetic disorder that causes nerve cells in the brain to break down. Over time, people with the disease can develop personality changes and have difficulty walking and swallowing.

Darzalex (daratumumab) is being proposed as a first-line treatment for newly diagnosed multiple myeloma in combination with three other cancer drugs for patients who are ineligible for an autologous stem cell transplant.