Regulation & Policy

The European Medicines Agency has recommended approval of an antibody-drug conjugate (ADC) for the treatment of metastatic triple-negative breast cancer, an aggressive cancer that does not respond to hormonal therapy medicines or medicines that target HER2 protein receptors. The positive opinion was based on data from a Phase 3 trial which showed that the therapy, Trodelvy (sacituzumab govitecan), produced an overall survival figure of 11.8 months and progression-free survival of 4.8 months, compared with 6.9 months and 1.7 months respectively, for chemotherapy.

The US Food and Drug Administration has approved the first allogeneic tissue product for the treatment of children born without a thymus, a condition known as congenital athymia. The product, Rethymic, is a donor derived product that is processed and cultured and implanted into patients to help reconstitute immunity in the absence of a thymus gland. The thymus is located behind the sternum and between the lungs, and is active until puberty. The gland secretes thymosin, a hormone necessary for T cell development and production.

CureVac NV has withdrawn its application for a marketing authorisation for its first Covid-19 vaccine from the European Medicines Agency, citing plans to focus instead on a second generation product that has shown an improved immune response in an animal study. The second generation vaccine, CV2CoV, is being developed with GlaxoSmithKline Plc. It encodes for the same protein as the company’s earlier vaccine but differs in the design of non-coding regions of the mRNA backbone.

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.

Xywav is already approved for excessive daytime sleepiness in patients with narcolepsy. The active ingredient of the drug is oxybate which is a controlled substance. Given its potential risks, Xywav will be subject to strict safety controls, the FDA said in an announcement on 12 August.