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
The MHRA has given formal approval for Redx to re-commence a Phase I/IIa trial of experimental cancer therapy RXC004. RXC004 is an oral porcupine inhibitor targeting the Wnt signalling pathway, with the Phase I/IIa trial on track to resume in the first half of 2019. Lisa Anson, chief executive of the firm, commented: “I am
Cynata Therapeutics, the Australian stem cell and regenerative medicine firm, has obtained approval from the UK MHRA to conduct its Phase 1 clinical trial of CYP-001 in patients with steroid-resistant graft-versus-host disease (GvHD). CYP-001 is Cynata’s lead Cymerus mesenchymal stem cell MSC product.
UK regulator, MHRA, has updated its guidance on whether a health app should be subject to medical device oversight. A variety of apps and stand-alone software that are already on the market have so far been classified as medical devices meaning that they fall within the MHRA’s remit.
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