NICE has today (13 March 2025)
recommended the first ever licensed treatment for an ultra-rare,
inherited immune system disorder for use in the NHS in
England.
NICE's final draft guidance recommends
leniolisib (also called Joenja and made by Pharming) for treating
activated phosphoinositide 3-kinase delta syndrome (APDS) in
people 12 years and over.
First recognised as a unique disease
in 2013, APDS is a rare, inherited, progressive 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.
PDS is caused by mutations in the
genes that are important for the growth, survival and function of
certain types of immune cells called T cells and B cells. These
are specialised white blood cells that help fight
infection.
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. 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 applying a QALY weighting. This increased the
threshold at which the treatment can be considered a
cost-effective use of NHS resources from £100,000 per QALY to
around £180,000.
The company also agreed a further
reduction to the £352,000 per year per patient list price of
leniolisib.
Helen Knight, director of
medicines evaluation at NICE, 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.”
Professor , 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, which underscores our enduring
commitment to securing innovative medicines for people affected
by rare conditions like this.”
Leniolisib is taken as a tablet and
works by blocking the phosphoinositide 3-kinase delta protein
that is overactive in APDS. By inhibiting the protein, leniolisib
helps normalise immune function as measured by an increase in
number of immune response generating B cells and a reduction in
size of lymph nodes.
- The draft guidance on leniolisib is available on the NICE
website at https://www.nice.org.uk/guidance/gid-hst10059/documents/html-content-8
