Three Investigational Therapies Get UK Innovation Passports

The Medicines and Healthcare products Regulatory Agency (MHRA) has awarded Innovation Passports to three investigational treatments for lung cancer, Huntington’s disease, and Alzheimer’s disease.The passports give the treatments access to the Innovative Licensing and Access Pathway (ILAP). The pathway provides coordinated support throughout development and is designed to reduce development risk and speed access to new medicines and drug-device combinations for UK patients.In a press release, the MHRA described ILAP as “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”.The three therapies show “real promise” in addressing the unmet needs in their respective diseases, said Professor Jacob George, the MHRA’s chief medical and scientific officer.Three Treatments, Three Conditions One Innovation Passport has been awarded to brezosiran, an siRNA (small interfering RNA) that can silence genes by cleaving mRNA in the treatment of Huntington’s disease. The rare, inherited, and ultimately fatal neurodegenerative disease affects around 8000 people in the UK. There were currently no therapies approved to slow its progression and new treatment options are urgently needed, said Dr Yvonne Greenstreet, chief executive officer of brezosiran’s developer, Alnylam.A second passport went to a PRMT5 (protein arginine methyltransferase 5) inhibitor, a small molecule oncology therapy. First-line non-small cell lung cancer (NSCLC) is expected to be its lead indication. NSCLC is the most common type of lung cancer, accounting for around 80%–85% of cases. Around 50,200 people are diagnosed with lung cancer in the UK each year. Despite advances in the treatment of NSCLC, “significant unmet needs remain”, said James Harris, interim general manager of the therapy’s developer, BeOne Medicines.The third Innovation Passport was granted to diranersen, an antisense oligonucleotide (ASO) for the treatment of early Alzheimer’s disease. Alzheimer’s disease remains “one of the greatest health and social care challenges of our time”, said Dr Kylie Bromley, managing director of the drug’s developer, Biogen UK and Ireland.A UK-Wide Partnership The ILAP is delivered through a partnership between the MHRA, NHS England, and the UK health technology assessment bodies – the National Institute for Health and Care Excellence, the Scottish Medicines Consortium (SMC), the All Wales Therapeutics and Toxicology Centre (AWTTC), and the Department of Health Northern Ireland. Partner organisations also welcomed the awards. The three conditions continued to place a “substantial burden” on patients, families, and healthcare systems, said Dr Rob Peel, chair of the SMC, in a press release. Dr Andrew Champion, AWTTC programme director, said that the award of Innovation Passports was an “encouraging step towards addressing unmet needs” of the three diseases.The three products are still in development, and any future availability will depend on the evidence of safety, effectiveness, and value, the MHRA said.Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.