Regulation & Policy

The US Food and Drug Administration has given an emergency use authorisation for a booster dose of the Pfizer/BioNTech Covid-19 vaccine for individuals 65 years and older and those who are at high risk of the disease. The high-risk population includes people who are healthcare workers and teachers, as well as those in homeless shelters and prisons.

The European Medicines Agency has issued a positive opinion recommending three new indications for Nucala (mepolizumab), a monoclonal antibody that targets interleukin-5 in order to treat a group of rare inflammatory diseases. The diseases all involve eosinophils, a type of white blood cell which can build up and cause inflammation.

The US Food and Drug Administration has reorganised its information technology work into a new office and appointed British educated Vid Desai to lead it. The new Office of Digital Transformation will combine all of the functions of the agency’s current IT, data management, and cybersecurity functions in a single unit, reporting directly to the FDA commissioner. It is part of a modernisation drive started in 2019 to operate more efficiently and increase the use of new technologies in the review of medicines and the monitoring food safety.

Jyseleca (filgotinib), a small molecule drug that blocks enzymes contributing to inflammatory diseases, is being recommended by the European Medicines Agency for the treatment of ulcerative colitis. Jyseleca has already been approved in the EU for rheumatoid arthritis. A new indication would broaden the drug’s coverage to include the treatment of adults with moderate to severely active ulcerative colitis who have not responded to earlier treatments.

The European Union and AstraZeneca Plc have reached a settlement on the supply of Vaxzevria, the company’s Covid-19 vaccine, to the union ensuring that vaccine deliveries through March 2022 will meet the terms of an advance purchase agreement concluded on 27 August 2020.

Valneva SE has started submitting regulatory data in support of its Covid-19 vaccine candidate to the UK Medicines and Healthcare products Regulatory Agency (MHRA) with a view to receiving an initial approval before the end of the year. The vaccine, VLA2001, is currently being studied in separate Phase 3 trials in the UK and New Zealand. Top-line results from the UK study are expected early in the fourth quarter.

The US Food and Drug Administration has approved a new indication for Xywav for the treatment of idiopathic hypersomnia, a chronic sleep disorder. The disorder causes people to be excessively sleepy during the day, even after a sound sleep the night before.

The accelerated approval pathway, a regulatory procedure used by the Food and Drug Administration to speed drugs to market that promise to treat serious medical conditions, is to be reviewed by the Office of Inspector General (OIG), the largest oversight body in the US government. Announced in early August, the review was triggered by the FDA’s accelerated approval on 7 June of Aduhelm, a treatment for Alzheimer’s disease. This decision led to the resignation of three members of an FDA advisory committee who argued that data presented in the drug’s application was inadequate.

A new enzyme replacement therapy for Pompe disease has been approved by the US Food and Drug Administration, which has been shown to help reduce glycogen accumulation in  muscles – a feature of the disease. The therapy, Nexviazyme (avalglucosidase), is a version of Myozyme (alglucosidase alfa), which was approved by the FDA in 2006. Both therapies were developed by Genzyme, which is part of Sanofi SA.

A new biologic for systemic lupus erythematosus has been approved by the US Food and Drug Administration, marking the first innovation in the treatment of the disease in more than a decade, the sponsor AstraZeneca Plc announced on 2 August. Saphnelo (anifrolumab) blocks a receptor in the type 1 interferon pathway preventing aberrant activation of the immune system. It is this activation that can cause immune cells to mistakenly attack healthy tissues in the body, a hallmark of lupus.