Regulation & Policy

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oversight over AstraZeneca’s latest Covid-19 vaccine trial, has expressed concern that the company may have included outdated information from the trial when it reported an efficacy rate of 79% for the vaccine on 22 March.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.

The European Medicines Agency has concluded that the benefits of the AstraZeneca/Oxford University Covid-19 vaccine outweigh the risks despite a possible link to rare blood clots with low blood platelets. The statement was issued on 18 March following a meeting of the agency’s Pharmacovigilance Risk Assessment Committee.

The European Commission has given a conditional marketing authorisation to the Covid-19 vaccine developed by Janssen Pharmaceutica NV following a positive scientific review by the European Medicines Agency. The decision, announced on 11 March, comes just under two weeks after the US gave the same vaccine an emergency use authorisation.

The European Medicines Agency is recommending approval of Evrysdi (risdiplam) to treat spinal muscular atrophy (SMA) – a rare and often fatal disease affecting neurons in the brain and spinal cord that control muscle movement. SMA is caused by mutations in the SMN1 gene that encodes for the survival motor neuron protein.

Merck & Co Inc has voluntarily withdrawn the metastatic small cell lung cancer indication for Keytruda (pembrolizumab) following the results of a Phase 3 study which didn’t support the terms of its earlier accelerated approval from the US Food and Drug Administration. The FDA awards accelerated approvals to certain drugs, based on surrogate endpoints, but confirmation of clinical benefit needs to be verified in subsequent trials.