Three experimental treatments win Innovation Passports, for lung cancer, Huntington's and early Alzheimer's
A gene-silencing therapy, a cancer drug aimed at tumours with no targeted treatment, and an Alzheimer's therapy have entered the UK's fast-support pathway. None is approved, and the regulator stresses that access will depend on evidence still to come.
Three medicines still in development have been given Innovation Passports by the Medicines and Healthcare products Regulatory Agency, the regulator announced on Monday. The awards admit the treatments to the Innovative Licensing and Access Pathway, known as ILAP, under which a developer works with the regulator, the health service and the bodies that assess value through one coordinated route, beginning well before a product is ready to be judged for use.
The selection panel's choices cover three very different kinds of therapy. One is a chemical small interfering RNA, a molecule designed to silence genes by cleaving the messenger RNA that carries their instructions, aimed at Huntington's disease. The second is a chemical small molecule cancer therapy, with first-line treatment of non-small cell lung cancer expected as its lead use. The third is an antisense oligonucleotide directed at early Alzheimer's disease.
The companies behind the awards named their own products in the announcement. Alnylam's chief executive, Yvonne Greenstreet, identified its RNA interference therapeutic as brezosiran, for Huntington's. BeOne Medicines named BGB-58067, an investigational PRMT5 inhibitor, and pointed to patients with MTAP-deficient tumours, for whom it said no targeted therapies are currently available; the award is the company's second into the pathway in recent months. Biogen's UK and Ireland managing director, Dr Kylie Bromley, welcomed the designation for diranersen in Alzheimer's disease.
Today, we have issued Innovation Passports to three therapies that show real promise in addressing the unmet needs in Alzheimer’s, lung cancer and Huntington’s disease.Professor Jacob George, Chief Medical and Scientific Officer, MHRA
The burden figures in the announcement explain the choices. Huntington's is described as a rare, inherited and ultimately fatal neurodegenerative disease. Non-small cell lung cancer is the most common form of lung cancer, making up around 80 to 85 per cent of cases, in a disease diagnosed in about 50,200 people in the UK each year. Alzheimer's is the most common cause of dementia: an estimated 982,000 people in the UK were living with dementia in 2024, and that total is expected to grow.
The assessment bodies were careful to frame the awards as a beginning. John Spoors, deputy director of medicines evaluation at NICE, said his organisation welcomed the chance to work with developers early on the questions that will shape later evaluation and access. Dr Rob Peel, chair of the Scottish Medicines Consortium, said in the announcement that the conditions involved place a substantial burden on patients, families and healthcare systems. Dr Andrew Champion, programme director of the Welsh assessment body, said his organisation would bring the needs of patients and NHS Wales into the shared process.
Nothing in the award changes what patients can receive today. The MHRA is explicit that the three products are still in development, and that whether any of them becomes available will rest on proof that it is safe, that it works and that it offers value. The pathway itself is delivered by the MHRA with NHS England, NICE, the Scottish Medicines Consortium, the Welsh therapeutics body and the Department of Health Northern Ireland. Applications for the next round of passports opened on Monday, 9 November, and remain open until 11 February 2027.
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