MHRA to allow for early access to Alnylam’s ultra-rare disease treatment, lumasiran

The MHRA has granted Alnylam’s lumasiran a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). The decision will allow patients with the ultra-rare disease primary hyperoxaluria type 1 (PH1) access to the drug before its official approval by the European Commission. PH1 is an ultra-rare orphan disease affecting around 90 patients in

Continue Reading

UK Launches Regenerative Medicines Regulatory Advice Service

A new ‘one-stop shop’ was launched in the UK yesterday, to provide regulatory advice for life science organisations working on regenerative medicines. The service, which will sit within the MHRA’s Innovation Office, will provide cross-regulatory advice from the regulatory bodies that work within regenerative medicines: the Human Tissue Authority; Health Research Authority; Human Fertilisation and

Continue Reading