Experimental Huntington’s drug selected for UK access pathway

An experimental drug for Huntington’s disease has been granted an Innovation Passport, giving its developer access to coordinated support from UK regulators, health services and medicines assessment bodies.

Brezosiran, developed by Alnylam, is one of three investigational treatments selected by the Medicines and Healthcare products Regulatory Agency (MHRA) for the UK’s Innovative Licensing and Access Pathway (ILAP).

The drug uses small interfering RNA, or siRNA, to silence gene activity by breaking down messenger RNA, which carries the instructions cells use to make proteins.

Huntington’s is a rare, inherited neurodegenerative disease that progressively affects movement, thinking and behaviour. There are currently no approved treatments that slow its progression.

The passport allows Alnylam to work with the MHRA and its partners during clinical development to identify what evidence will be needed for future assessments. Any decision on whether to make brezosiran available to patients will depend on evidence of its safety, effectiveness and value.

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.

“We are honored that our investigational RNAi therapeutic brezosiran has received an Innovation Passport and welcome the opportunity to work closely with the MHRA and ILAP partners as we advance its development.”

John Spoors, deputy director of medicines evaluation at NICE, said the pathway would give the organisation an opportunity to work with developers early on the evidence needed to inform future evaluation and patient access.

The MHRA also awarded passports to BeOne Medicines’ BGB-58067, which is being developed for non-small cell lung cancer, and Biogen’s diranersen, an investigational treatment for early Alzheimer’s disease.