Regulation & Policy

An antibody treatment for the rare autoimmune disorder, neuromyelitis optica spectrum disorder, has received a positive opinion from the European Medicines Agency following two studies showing that the therapy reduced the chance of disease relapse. The treatment, Enspryng (satralizumab), was developed by the Roche Group. It targets the interleukin-6 receptor which is believed to play a role in the inflammation associated with the disease.

Enspryng was reviewed for adults and adolescents who are positive for the pathogenic antibodies, AQP4, an indicator of the disease.

A new drug for endometrial cancer has been approved by the US Food and Drug Administration following a trial which showed an overall response rate of 42.3%. Jemperli (dostarlimab) was developed by GlaxoSmithKline Plc and is administered with a diagnostic developed by Roche. The diagnostic can recognise cancers which are deficient mismatch repair (dMMR), or cancers with a genetic feature that adversely affects the repair of DNA inside cells.

Johnson & Johnson Inc is to resume the rollout of its single dose vaccine for Covid-19 in Europe following guidance from the European Medicines Agency that the vaccine’s benefits outweigh its risks. Eight cases of unusual blood clots associated with low levels of blood platelets have been identified in the US, one of which was fatal. But set against this, are the more than seven million people who have received the vaccine without serious incident, officials from the EMA said on 20 April, following a safety review.

Opdivo (nivolumab), one of the earliest checkpoint inhibitors, has been approved by the US Food and Drug Administration for the first-line treatment of gastric cancer – the first immunotherapy for this indication. The approval also covers gastroesophageal junction cancer and oesophageal adenocarcinoma.

The first medical device to use artificial intelligence to detect colon cancer has been approved by the US Food and Drug Administration. The device, GI Genius, was developed by Cosmo Artificial Intelligence Ltd and has shown an ability to detect lesions in the colon in real time during a colonoscopy. In a statement issued on 10 April, the FDA’s Courtney Lias said that “clinicians now have a tool that could help improve their ability to detect gastrointestinal lesions they may have missed otherwise.”

Sarclisa (isatuximab), a monoclonal antibody developed by Sanofi SA, has been approved for a second multiple myeloma indication in the US after showing that it reduced the risk of disease progression or death by 45% compared with the standard of care.

The US Food and Drug Administration has approved a cell-based gene therapy for multiple myeloma – the first therapy of its type for myeloma, an uncommon type of blood cancer. Abecma (idecabtagene vicleucel) is a chimeric antigen receptor (CAR) T cell therapy targeting the B cell maturation antigen (BCMA), a cell membrane receptor expressed exclusively on late stage B cells and plasma cells. Abecma has been approved for adult patients who have not responded to, or whose disease has returned after, at least four prior lines of therapy.

A new endocrine treatment for patients with congenital adrenal hyperplasia is being recommended for marketing in Europe after achieving positive results in a Phase 3 study of 122 patients with the disease. The European Medicines Agency delivered the positive opinion on 26 March, ahead of a decision on marketing authorisation by the European Commission.

In a bid to secure an adequate supply of Covid-19 vaccines for the European population, the European Commission has tightened a regulation, introduced at the start of the year, to monitor vaccine exports from the EU to other countries in the world. The move has generated concern from industry groups worried about a possible disruption to vaccine supply chains. However during a press briefing on 24 March, the Commission said the goal of the regulation was to achieve a “security of supply” for Europe while maintaining the integrity of the international trading system.

An independent data and safety monitoring board (DSMB), which had oversight over AstraZeneca’s latest Covid-19 vaccine trial, has expressed concern that the company may have included outdated information from the trial when it reported an efficacy rate of 79% for the vaccine on 22 March. The DSMB concern was cited in a press release issued by the National Institute of Allergy and Infectious Diseases (NIAID), an agency of the US National Institutes of Health, on 23 March.