The European Medicines Agency has given a positive opinion to a new gene therapy for cerebral adrenoleukodystrophy (CALD), a rare inherited neurological disease that affects approximately one in 21,000 newborn males. The scientific endorsement, which is the first step towards a formal marketing authorisation, is based on data from a single-arm trial.
The US Food and Drug Administration has approved a new bispecific antibody for the treatment of non-small cell lung cancer whose tumours have a specific genetic mutation. Rybrevant (amivantamab) was authorised for patients with lung cancers which have an epidermal growth factor receptor (EGFR) exon 20 insertion mutation.
GlaxoSmithKline Plc and its US partner Vir Biotechnology Inc have submitted a candidate antibody for patients at risk of severe Covid-19 disease to the European Medicines Agency for a rolling review. A rolling review is a procedure by which a regulator examines data for a prospective drug as it becomes available, rather than waiting until all clinical trials are complete. This enables medicines that are important to public health to be reviewed more quickly.
A new antibody-drug conjugate (ADC) has received US approval to treat patients with a type of non-Hodgkin lymphoma after showing an overall response rate of 48.3% in a pivotal Phase 2 trial. Zynlonta (loncastuximab tesirine) was authorised by the Food and Drug Administration as a single-agent treatment for diffuse large B cell lymphoma (DLBCL) – the first single-agent ADC for this disease. Patients in the trial had already received two or more lines of systemic therapy, but these treatments had failed.
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.