Regulation & Policy

A new intravenous treatment for the prevention of migraine has been approved by the US Food and Drug Administration on the basis of trial data showing that patients had a sustained reduction in their disease episodes for a period of months after receiving the drug. The treatment, Vyepti (eptinezumab), is a monoclonal antibody that binds to the human calcitonin gene-related peptide with the result that migraine attacks are reduced. It was developed by Alder BioPharmaceuticals Inc, a company that was acquired by H. Lundbeck A/S of Denmark in 2019.

The first gene therapy for haemophilia to be submitted for regulatory review, valoctocogene roxaparvovec, has been given ‘priority review’status by the US Food and Drug Administration. Developed by BioMarin Pharmaceutical Inc, the therapy will be assessed for safety and efficacy for the treatment of patients with haemophilia A. Haemophilia A is the more prevalent of the two disease types, haemophilia A and B. Under a priority review, the FDA aims to issue a decision on marketing in six months rather than the usual 10, which means the outcome of the review should be known by 21 August.

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.