MHRA Backs Three New Treatments for Serious Diseases

The UK medicines regulator has awarded Innovation Passports to three experimental treatments for lung cancer, Huntington's disease and early Alzheimer's disease. The Medicines and Healthcare products Regulatory Agency (MHRA) announced the awards on 9 November 2026, giving the developers coordinated support to bring the treatments through development more quickly.
The three treatments were chosen by the Innovative Licensing and Access Pathway (ILAP) Selection Panel because they address areas of serious unmet medical need. None of the three is yet approved, and none is available to patients.
What the three treatments target
The first is brezosiran, an experimental gene-silencing treatment developed by Alnylam for Huntington's disease. It is a small interfering RNA therapy designed to switch off specific genes by breaking down messenger RNA. Huntington's disease is a rare, inherited and ultimately fatal neurodegenerative condition, and there are currently no approved therapies that slow its progression.
The second is BGB-58067, a small-molecule cancer medicine developed by BeOne Medicines, with first-line non-small cell lung cancer expected to be its lead use. It is a PRMT5 inhibitor being investigated for patients with MTAP-deficient tumours, for whom no targeted therapies are currently available. Non-small cell lung cancer is the most common type of lung cancer, accounting for around 80 to 85 per cent of cases, and around 50,200 people are diagnosed with lung cancer in the UK each year.
The third is diranersen, an antisense oligonucleotide treatment developed by Biogen for early Alzheimer's disease. Alzheimer's is the most common cause of dementia, and an estimated 982,000 people were living with dementia in the UK in 2024.
What an Innovation Passport means
An Innovation Passport gives developers access to the ILAP, a single platform where they can work from an early stage with the regulator, the NHS and the bodies that assess value for money. The pathway is delivered in partnership with the MHRA, NHS England, the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium, the All Wales Therapeutics and Toxicology Centre and the Department of Health in Northern Ireland.
Professor Jacob George, the MHRA's chief medical and scientific officer, said the three therapies show real promise in addressing unmet needs in Alzheimer's, lung cancer and Huntington's disease. The MHRA stressed that the products remain in development and that any future availability will depend on evidence of safety, effectiveness and value.
Applications for the next round of Innovation Passports opened on 9 November 2026 and remain open until 11 February 2027. Developers of innovative medicines that address unmet patient needs are invited to apply. Read the full announcement on GOV.UK and the summary from Open Access Government.