Daijiworld Media Network - London
London, Nov 10: The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has awarded Innovation Passports to three investigational treatments targeting lung cancer, Huntington's disease and Alzheimer's disease, providing them access to a special pathway designed to accelerate medicine development and patient access.
The passports allow the treatments to enter the Innovative Licensing and Access Pathway (ILAP), which offers coordinated support throughout the development process and aims to reduce risks and speed up access to new medicines and drug-device combinations for patients in the UK.
In a statement, the MHRA described ILAP as the only end-to-end access pathway in the world through which developers can work with the national health system, regulator and health technology assessment bodies from the early stages of clinical development.
The three therapies show "real promise" in addressing unmet medical needs associated with their respective conditions, said Professor Jacob George, the MHRA's chief medical and scientific officer.
One Innovation Passport was awarded to brezosiran, a small interfering RNA (siRNA) therapy being developed by Alnylam for Huntington's disease. The treatment works by silencing specific genes through the cleavage of messenger RNA.
Huntington's disease is a rare, inherited and ultimately fatal neurodegenerative condition affecting around 8,000 people in the UK. There are currently no approved therapies that can slow its progression, making new treatment options urgently necessary, said Dr Yvonne Greenstreet, chief executive of Alnylam.
The second passport was granted to a protein arginine methyltransferase 5 (PRMT5) inhibitor, a small-molecule cancer therapy being developed by BeOne Medicines. Its lead proposed indication is first-line treatment for non-small cell lung cancer (NSCLC), the most common form of lung cancer, accounting for around 80 to 85 per cent of cases.
Around 50,200 people are diagnosed with lung cancer in the UK every year. Despite advances in NSCLC treatment, significant unmet medical needs remain, said James Harris, interim general manager of BeOne Medicines.
The third Innovation Passport was awarded to diranersen, an antisense oligonucleotide (ASO) being developed by Biogen for early Alzheimer's disease.
Alzheimer's disease remains one of the greatest challenges facing health and social care systems, said Dr Kylie Bromley, managing director of Biogen UK and Ireland.
The ILAP is delivered through a partnership involving the MHRA, NHS England and UK health technology assessment bodies, including the National Institute for Health and Care Excellence, the Scottish Medicines Consortium, the All Wales Therapeutics and Toxicology Centre, and the Department of Health in Northern Ireland.
Welcoming the awards, Dr Rob Peel, chair of the Scottish Medicines Consortium, said the three conditions continued to place a substantial burden on patients, families and healthcare systems.
Dr Andrew Champion, programme director at the All Wales Therapeutics and Toxicology Centre, described the passports as an encouraging step towards addressing the unmet needs associated with the diseases.
The MHRA emphasised that all three products remain under development. Any future availability will depend on evidence demonstrating their safety, effectiveness and value.