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.
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.
The European Medicines Agency has given a positive scientific review to a new treatment for achondroplasia, a condition that impairs bone growth and causes dwarfism. It is recommending that a marketing authorisation be issued to BioMarin Pharmaceutical Inc, the developer, for use of Voxzogo (vosoritide) in patients two years and older whose epiphyses, or growth plates of the bones, are not yet closed. This condition must be confirmed by genetic testing. The medicine was evaluated in 121 patients with achondroplasia between the ages of five and 18 years.
Janet Woodcock, acting commissioner of the US Food and Drug Administration, has asked federal officials to conduct an investigation of interactions between agency staff and executives at Biogen Inc in the process that led up to the FDA’s approval of Aduhelm (aducanumab) for Alzheimer’s disease on 7 June. A letter was sent to the acting inspector general of the Department of Health and Human Services on 9 July and distributed globally on the same day on Twitter.
A gene therapy for multiple myeloma that was approved in the US in March has now been recommended for a conditional marketing authorisation by the European Medicines Agency. 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.