Regulation & Policy

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.

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.

With Brexit looming, the European Medicines Agency now believes that it could lose up to 30% of its staff as the agency prepares to move from its current location in London to new premises in Amsterdam. The move is scheduled to take place in March 2019.

As an agency of the European Union, the EMA must be located in an EU member state. London will no longer be eligible after Britain leaves the EU which is set for 23.00 BST on 29 March 2019.

An RNA therapeutic developed by Alnylam Pharmaceuticals Inc is the newest drug to be recommended for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a life-threatening disease caused by mutations in the transthyretin (TTR) gene.