MHRA grants Innovation Passports to three novel drugs
gov.uk
The UK medicines regulator has awarded Innovation Passports to three medicines that could offer a step forward in the treatment of lung cancer, Huntington's disease, and Alzheimer's.
The coveted status – which is designed to speed up NHS access to promising new medicines – has gone to BeOne Medicines' brain-penetrant PRMT5 inhibitor BGB-58067 for non-small cell lung cancer (NSCLC), Alnylam/Regeneron's HTT-targeting brezosiran for Huntington's, and Biogen/Ionis' diranersen, a tau protein inhibitor for Alzheimer's.
Being awarded an Innovation Passport means the medicines will be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA) under the innovative licensing and access pathway (ILAP) introduced in 2020, which reduces the review time for a new medicine to 150 days and gives the sponsor company additional support.
"This is the only end-to-end access pathway in the world where developers can work, from early stages of clinical development, with the national health system, the regulator and health technology assessment bodies," said the MHRA in a statement.
"The ILAP is designed to de-risk product development and accelerate patient access to transformative new medicines and drug-device combinations by providing a single integrated platform for collaboration."
BGB-58067 is designed to selectively target tumour cells with methylthioadenosine phosphorylase (MTAP) deficiency, found in approximately 10%-15% of tumours across multiple cancer types, and has shown promising preliminary activity in a phase 1 trial reported at this year's ESMO cancer congress. BeOne has said it plans to start an international registration trial of BGB-58067 in MTAP-deficient NSCLC in 2027.
Brezosiran (formerly ALN-HTT02) is currently in a phase 1b trial for Huntington's disease, with a pivotal trial planned for 2027, according to Alnylam. The drug switches off exon 1 of the huntingtin (HTT) gene and is designed to switch off the production of neurotoxic forms of the huntingtin protein that accumulate in the disorder. It is administered via intrathecal injection with a projected twice-yearly dosing schedule.
Diranersen, meanwhile, has completed the phase 2 CELIA trial, which missed its primary endpoint but showed signs of cognitive benefit, and is being prepared to advance into a phase 3 development programme. It has been administered via intrathecal injection at intervals of every three or six months in clinical trials, and shuts down the production of tau – a protein that clumps together in the neurofibrillary tangles that are one of the hallmarks of Alzheimer's.
ILAP works similarly to the EU's Priority Medicines (PRIME) pathway, which also provides a fast-track review process, but has an additional dimension as it also includes funding considerations through the involvement of NICE, the Scottish Medicines Consortium (SMC) and the All Wales Therapeutics and Toxicology Centre (AWTTC).
