Regulation & Policy

New confirmed cases of infections from the coronavirus in China, also known as COVID-19, are “generally in decline,” China’s ambassador to the EU, Zhang Ming, told journalists on Tuesday. The ambassador was speaking at a meeting in Brussels, organised by the think tank EU-Asia. Topics included EU-China trade relations, but the virus epidemic dominated the conversation. Mr Ming was at pains to detail China’s efforts to contain the epidemic and predicted that the contagion would soon be overcome.

The US Food and Drug Administration has issued new guidance for developers of gene therapies highlighting both the complexity of manufacture as well as the difficulty in establishing duration of response in clinical trials.

In six final guidances, the agency addresses these issues as well as the application of orphan drug regulations to gene therapy. Orphan drug eligibility is an issue because many gene therapies are being developed for rare diseases where the patient populations are small.

The European Medicines Agency has won a case in the Court of Justice of the European Union upholding its right to publicly disclose information from a pharmaceutical company’s application for marketing, once that product has been approved. The case concerns a decision by the EMA to grant a third party access to a regulatory dossier of PTC Therapeutics International Ltd in 2015 for a product for Duchenne muscular dystrophy.

A new epigenetic medicine has been approved in the US to treat epithelioid sarcoma, a rare sub-type of soft tissue sarcoma that affects young adults. The drug, Tazverik (tazemetostat), is a small molecule inhibitor of EZH2 methyltransferase which regulates gene expression. By blocking the activity of EZH2, the drug is expected to keep cancer cells from growing. The Food and Drug Administration announced the authorisation on 23 January.

A new drug has been approved in the US for thyroid eye disease, a rare condition in which the muscles and fatty tissues behind the eye become inflamed, causing the eyes to bulge. Tepezza (teprotumumab) is a monoclonal antibody that targets the insulin-like growth factor-1 receptor with the goal of modifying the course of the disease. In issuing the approval, the Food and Drug Administration said Tepezza is the first non-surgical treatment for thyroid eye disease, which can cause eye pain, double vision and sensitivity to light.

Lynparza (olaparib) has been approved in the US as a first-line maintenance treatment for metastatic pancreatic cancer, the first therapy of its type to show a statistically significant improvement in progression-free survival in patients with the disease. The Food and Drug Administration announced the approval on 30 December on the basis of data from a Phase 3 trial of 154 patients who had germline BRCA-mutated pancreatic cancer.

Patients will be eligible for the drug based on an FDA-approved companion diagnostic.

Business hubs where life science companies co-locate in order to share infrastructure exist almost everywhere. But not all business hubs support basic science, let alone national healthcare systems. EIT Health is different. The group is part of the European Institute of Innovation and Technology which was set up in 2015 to promote the interface between industry, science and public healthcare systems. At conference in Paris on 2 to 3 December, members of the consortium met to discuss issues facing healthcare – and to congratulate a new crop of entrepreneurs.

The US Food and Drug Administration has approved a new drug for Duchenne muscular dystrophy (DMD) on the basis of data showing that the medicine increased the production of  the dystrophin protein in patients – a surrogate for improved motor function. The drug, Vyondys 53, was developed by Sarepta Therapeutics Inc, whose first DMD drug Exondys 51 was approved by the FDA in 2016, also on the basis of a surrogate endpoint.

The first vaccine for the active immunisation of individuals at risk of infection from the Ebola virus has been cleared by the World Health Organization for procurement by the United Nations and included in a stockpile for use in countries affected by the disease. These countries include the Democratic Republic of Congo where a public health emergency has been declared by the WHO.

A new drug for anaemia in patients with beta thalassaemia, a rare blood disorder, has been approved for marketing in the US following clinical testing which showed that patients receiving the treatment needed fewer blood transfusions than those on a placebo. The drug, Reblozyl (luspatercept-aamt), was authorised by the Food and Drug Administration on 8 November.