A drug developed by UCL researchers and granted orphan drug designation is one of the first to take part in the newly launched adaptive licensing pilot project to speed up patients’ access to new medicines.
The drug is the first treatment aimed at directly removing amyloid deposits from the tissues of people with the rare and usually fatal disease, systemic amyloidosis.
Developed by Professor Sir Mark Pepys FRS, Director of the UCL Wolfson Drug Discovery Unit, the new treatment involves an obligate therapeutic partnership between a small molecule drug, CPHPC and a monoclonal antibody. The first clinical trial began in 2013 and continues to deliver encouraging results, showing clearance of amyloid deposits.
Amyloidosis is a group of rare but serious conditions caused by abnormal protein deposits, called amyloid, in tissues and organs throughout the body. Unlike normal proteins, the body does not easily break down amyloid. The deposits build up in tissues and organs, damaging their structure and function, leading to disease.
Adaptive licensing was launched this year by the European Medicines Agency to improve timely access for patients to new medicines. It is a prospectively planned process, starting with the early authorisation of a medicine in a restricted patient population, followed by evidence gathering and adaptations of the marketing authorisation in order to expand access to the medicine to broader patient populations.
Orphan drug designation by the European Medicines Agency is aimed at encouraging the development of treatments for rare diseases. Orphan drug designation brings various advantages including 10 year market exclusivity
Sir Mark has worked on amyloidosis since 1976 and in the 1980s invented SAP scintigraphy for diagnosis and monitoring of the disease. He established, with Professor Philip Hawkins, the UK NHS National Amyloidosis Centre in 1999. In 2005 Sir Mark invented a new approach to treatment of systemic amyloidosis, involving an obligate therapeutic partnership between a small molecule drug, CPHPC, which he had previously developed with Roche, and a monoclonal antibody against the protein SAP which is always present in amyloid deposits. In 2009 the invention was licensed to GlaxoSmithKline by the UCL spinout company, Pentraxin Therapeutics Ltd founded by Sir Mark.
Sir Mark established the Wolfson Drug Discovery Unit in 2011 supported by a £2 million capital award from the Wolfson Foundation and BRC funding for the operations of the unit.
