First licensed treatment for ultra-rare immune disorder recommended
Leniolisib is the first ever treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) licensed for use in the NHS in England.
In final draft guidance published today, we have recommended leniolisib (also called Joenja and made by Pharming) for treating activated phosphoinositide 3-kinase delta syndrome (APDS), an ultra-rare inherited immune system disorder, in people 12 years and over.
First recognised as a unique disease in 2013, APDS is a condition that affects the way the immune system (the body’s natural defences) works. This means that people with APDS have a reduced ability to fight infections.
APDS can severely affect the quality of life of people with the condition, and their families and carers, and can significantly shorten life, with an average life expectancy of 44 years.[RS1] It can cause organs and lymph nodes to swell and the body’s immune system to attack healthy tissue. People with the condition are also at high risk of serious infections, and it can increase the risk of cancer.
Today’s decision follows the provision of further data by the company. This addressed the areas of uncertainty particularly around the effects of stopping treatment that were identified by the independent committee.
Addressing these uncertainties meant the committee was able to recognise the important benefits of leniolisib by increasing the threshold at which the treatment can be considered a cost-effective use of NHS resources.
The company also agreed a further reduction to the £352,000 per year per patient list price of leniolisib.
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.
Helen continued: "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."
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.
Professor Palmer continued: “The NHS is the first health system in Europe to offer this NICE approved treatment, for a condition identified just over a decade ago, which underscores our enduring commitment to securing innovative medicines for people affected by rare conditions like this.”