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'Next generation' precision medicine for cystic fibrosis wins prestigeous drug discovery award

Published: 14 Dec 2016 in Society news

A new medicine to treat cystic fibrosis has received Drug Discovery of the Year 2017, a prestigious award from the British Pharmacological Society. A combination of two drugs (lumacaftor and ivacaftor) offers a significant step forward for the treatment of this life-limiting, inherited disease. The award is made to the team of researchers at pharmaceutical company Vertex Pharmaceuticals, which is responsible for the development of lumacaftor/ivacaftor, in recognition of the drug offering an innovative approach that addresses a significant unmet medical need.
Cystic fibrosis is a genetic disease that can cause the lungs and digestive system to become clogged with thick, sticky mucus. Over time, the lungs become increasingly damaged and prone to chronic infection, and they may eventually stop working properly. A number of treatments are available to help reduce symptoms, but unfortunately average life expectancy is reduced for people with cystic fibrosis.
It is estimated that there are over 70,000 people with cystic fibrosis worldwide[i], and 1 in every 2,500 babies born in the UK has the condition[ii]. Children with cystic fibrosis inherit a defective or missing protein – known as CFTR – resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in this single gene.
Lumacaftor/ivacaftor is an example of precision medicines: it responds to one type of CFTR gene mutation in patients with cystic fibrosis — treating the root cause of the condition, rather than just focusing on symptoms. Patients in clinical trials experienced improvements in lung function and body mass index (BMI) scores. Lumacaftor/ivacaftor was approved for use in people over 12 years old with this gene mutation in the USA and Europe in 2015.
“By tackling the underlying causes of the disease, lumacaftor/ivacaftor marks progress in the development of the next generation of treatments for cystic fibrosis. The British Pharmacological Society is pleased to make this award to the team at Vertex Pharmaceuticals for their contribution to our understanding of precision medicines in the treatment of cystic fibrosis,” said Dr Tom Blackburn, chair of the British Pharmacological Society’s judging panel.
He added, “The British Pharmacological Society celebrates the importance of pharmacology in drug discovery and the benefit of this research to society. The life expectancy for someone with cystic fibrosis was 18 years old in the 1980s, but even in 2016 around half of patients die before their 41st birthday. Life expectancy will only continue to improve as the pharmacology community makes further advances in the treatments available to people with this life-limiting condition.”
James Barrow, Head of External Affairs at the Cystic Fibrosis Trust, commented: "The drug is an exciting breakthrough in the treatment of cystic fibrosis. It's the first time that two compounds have worked together to improve the function of the most common CFTR mutation. It is already transforming the lives of thousands people living with cystic fibrosis.”
The Drug Discovery of the Year award is being presented by Professor David Webb, President of the British Pharmacological Society, to the Vertex Pharmaceuticals team during the Society’s annual conference, Pharmacology 2016.

[i] Sosnay PR et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet. 2013;45(10):1160-1167.
[ii] NHS Choices. Cystic fibrosis. Available online: