The European Medicines Agency has recommended the conditional approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for haemophilia A. If approved, it would be the first gene therapy for this disease in Europe. The conditional approval carries with it a requirement that the developer, BioMarin Pharmaceutical Inc, provide follow-up data from ongoing clinical trials to show the therapy continues to be safe and efficacious for patients.
Regulation & Policy
As part of its strategy to incorporate big data into regulatory decision-making, the European Medicines Agency is to invite pharma companies to apply for a pilot project under which they would submit patient data from clinical trials as part of their marketing authorisation applications. The project is expected to start in September and last up to two years. It would cover about 10 regulatory procedures.
A second booster dose of one of the two messenger RNA (mRNA) vaccines approved in the EU for Covid-19 is being recommended for adults between the ages of 60 and 79 years. This is to protect against a new wave of infections caused by a variant of the SARS-CoV-2 virus. The recommendation was issued on 11 July by the European Centre for Disease Prevention and Control and the European Medicines Agency.
Kimmtrak (tebentafusp), a bispecific protein for the treatment of uveal melanoma, has received three more regulatory approvals on the back of clinical data showing that it extends overall survival in patients with the rare and aggressive form of melanoma. Approvals have been issued by regulatory authorities in the UK, Canada and Australia, the developer Immunocore Holdings Plc announced on 8 June. This follows approvals earlier in the year in the US and EU.
An enzyme replacement therapy for two types of Niemann-Pick disease is being recommended for marketing by the European Medicines Agency. If formally approved by the European Commission, the drug, Xenpozyme (olipudase alfa), would be the first specific treatment for the disease, which is a rare genetic metabolic disorder.
Priorix, a vaccine developed by GlaxoSmithKline Plc, has been approved by the US Food and Drug Administration for active immunisation for the prevention of measles, mumps and rubella (MMR) infection in individuals 12 months of age and older. The vaccine is currently licensed in more than 100 countries, including the nations of Europe, Canada, Australia and New Zealand but until now, has not been available in the US.
The US Food and Drug Administration has approved a new indication for Dupixent (dupilumab), broadening its role as a leading anti-inflammatory medicine for allergic diseases. The new use is for eosinophilic oesophagitis (EoE), a chronic inflammatory disorder in which eosinophils, a type of white blood cell, are found in the tissue of the oesophagus. This can impair a person’s ability to swallow. Dupixent is a monoclonal antibody that blocks interleukin 4 and interleukin 13 – part of the inflammatory pathway.
The US Food and Drug Administration has approved a new diabetes medicine which targets two hormones involved in blood sugar control. In clinical studies, Mounjaro (tirzepatide) was shown to be more be more effective than three other approved diabetes drugs. It was developed by Eli Lilly and Co and given an expedited review by the FDA on the basis of its potential for significantly improving the treatment of Type 2 diabetes. It is also under regulatory review in Europe and Japan.
The European Medicines Agency has created a new forum to discuss the way cancer drugs are developed and administered in order to improve the healthcare outcomes for patients. The forum was launched in late March in partnership with the European Organisation for Research and Treatment of Cancer (EORTC), a non-profit organisation located in Belgium,
The US Food and Drug Administration has given an accelerated approval to Novartis for a new drug to treat a rare group of genetic diseases known as PIK3CA-related overgrowth spectrum (PROS). The drug, Vijoice (alpelisib), was approved on the basis of real-world evidence from patients who received alpelisib as part of a compassionate use programme.