Experimental drugs for Huntington's, Alzheimer's and lung cancer given fast-track Innovation Passports

Three experimental medicines aimed at Huntington's disease, Alzheimer's disease and a common form of lung cancer have been given "Innovation Passports" by the UK medicines regulator, giving them a faster, joined-up route through development towards patients.

The Medicines and Healthcare products Regulatory Agency (MHRA) announced the awards on 9 November. The passports admit the treatments to the Innovative Licensing and Access Pathway (ILAP), which the regulator describes as the only end-to-end access pathway in the world where developers can work with the national health system, the regulator and the bodies that assess value for money, from early clinical development onwards.

The three treatments

The first is brezosiran, a small interfering RNA (siRNA) medicine from Alnylam for Huntington's disease. siRNA drugs work by silencing specific genes. Huntington's is a rare, inherited and ultimately fatal disease of the brain, and there are no approved treatments to slow its progression.

The second is BGB-58067, a cancer drug from BeOne Medicines that is expected to be tested first in non-small cell lung cancer, which makes up around 80 to 85% of lung cancers. Around 50,200 people are diagnosed with lung cancer in the UK each year. The drug targets tumours with a specific genetic change known as MTAP deficiency, for which there are currently no targeted therapies.

The third is diranersen, an antisense oligonucleotide from Biogen for early Alzheimer's disease, the most common cause of dementia. An estimated 982,000 people were living with dementia in the UK in 2024.

Why it matters

New medicines can take many years to move from the laboratory to the clinic. The pathway is designed to reduce the risk and delay by bringing the developer, the regulator, NICE and the NHS together early, so questions about evidence, safety and value are tackled along the way rather than at the end.

Professor Jacob George, the MHRA's Chief Medical and Scientific Officer, said: "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."

Yvonne Greenstreet, chief executive of Alnylam, said: "Huntington's disease is a devastating condition with no therapies approved to slow its progression, underscoring the urgent need for new treatment options."

Dr Kylie Bromley, managing director of Biogen UK and Ireland, said: "Alzheimer's disease remains one of the greatest health and social care challenges of our time."

A note of caution

A passport is not an approval. All three products are still in development, and the MHRA stresses that any future availability will depend on evidence of safety, effectiveness and value. But for families living with these conditions, it is a sign that promising science is being given a clearer road to the clinic.

Next round open now

Applications for the next round of Innovation Passports opened on 9 November and close on 11 February 2027. The MHRA is encouraging companies developing medicines for unmet needs in any area to apply. Birmingham has a growing life sciences sector, with research hospitals, universities and companies working on new treatments, and the scheme is open to developers of every size.