The first treatment for an inherited immune system disorder first identified in 2013 will be made available to patients in England, it has been announced.
Leniolisib will be available for patients over the age of 12 with activated phosphoinositide 3-kinase delta syndrome (APDS).
The National Institute for Health and Care Excellence (NICE) says the decision will help up to 50 people. The drug blocks the phosphoinositide 3-kinase delta protein that is overactive in the disease. This should reduce the size of lymph nodes and increase the number of B cells.
The list price of the drug is £352,000 a year, but NICE said the developers have agreed a discount for the NHS.
Professor James Palmer, NHS medical director for specialised commissioning, said: “I’m delighted that the NHS in England will provide a first-ever targeted treatment for adolescents and adults with this rare, genetic condition which weakens an individual’s immune system and can lead to serious issues and hospitalisation.
“The NHS is the first health system in Europe to offer this NICE approved treatment, for a condition identified just over a decade ago.”
NICE director of medicines evaluation Helen Knight said: “This is a significant milestone for people with this debilitating genetic condition who will now be able to access this first of its kind treatment routinely on the NHS.
“It is estimated that between 40 and 50 people have APDS and today’s decision means leniolisib will help give some of them and their families real hope of a better quality of life. We’re therefore pleased the company was able to work constructively with us to address the areas where more evidence was needed for NICE to be able to recommend this promising new treatment.”
Source: NICE
Disclaimer: The news stories shared on this site are used as a way to inform our members and followers of updates and relevant information happening in Haematology. The BSH does not endorse the content of news items from external sources, and is not in a position to verify the findings, accuracy or the source of any studies mentioned. Any medical or drugs information is provided as an information resource only, and is not to be relied on for any diagnostic or treatment purposes.
News service provided by Englemed News.