The US Food and Drug Administration has extended a clinical hold on a proposed Phase 3 clinical trial of a microbiome-based therapeutic developed by MaaT Pharma SA of France because it requires more information about the composition of the faecal-derived product. MaaT Pharma announced the clinical hold extension on 10 August.
Regulation & Policy
The US Food and Drug Administration has approved Enhertu (trastuzumab deruxtecan) for a new breast cancer indication while at the same time reclassifying a breast cancer subtype uniquely treated by the drug. Enhertu is an antibody-drug conjugate that combines a monoclonal antibody conjugated to the chemotherapy deruxtecan. The drug binds to a receptor on cancer cells after which it releases the antibody and toxin into cells to kill the cancer. Enhertu is being developed and commercialised by UK-based AstraZeneca Plc and Daiichi Sankyo Co Ltd of Japan.
Pharming Group NV is to get an accelerated review from the European Medicines Agency for leniolisib, its candidate treatment for a rare primary immunodeficiency known as activated phosphoinositide 3-kinase delta syndrome (APDS). A marketing authorisation application is expected to be submitted to the EMA in October, the company announced on 1 August.
The US Food and Drug Administration has launched a plan to support the development of new medicines for rare neurodegenerative diseases including amyotrophic lateral sclerosis, a progressive disease that affects nerves in the brain and spinal cord, causing loss of muscle control. The plan has been developed in line with new US legislation, the Accelerating Access to Critical Therapies for ALS Act that took effect on 23 December 2021.
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.
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.