The National Institute for Health and Care Excellence (NICE) has recommended Pharming’s Joenja (leniolisib) to treat patients with the ultra-rare immune disease, activated phosphoinositide 3-kinase delta syndrome (APDS).
The small molecule phosphoinositide 3-kinase delta inhibitor, which is now the first ever APDS treatment to be licensed for use in the NHS in England, has been indicated for use in adults and adolescents aged 12 years and older.
Approximately 40 to 50 people in the UK are known to be living with APDS, an inherited disorder that affects how the body’s immune system works, meaning patients have a reduced ability to fight infections.
The main symptoms usually occur in the first two years of life and include repeated lung infections, as well as a failure to grow and develop normally. Patients are also at an increased risk of developing blood cell cancers, such as lymphoma.
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 [this] decision means [Joenja] will help give some of them and their families real hope of a better quality of life.”
NICE’s final draft guidance follows the Medicines and Healthcare products Regulatory Agency’s approval of the drug in September and was supported by results from a 12‑week placebo-controlled study of 31 APDS patients.
By day 85 of the trial, patients randomised to receive Joenja saw a reduction in lymph node size and a 37% improvement in naïve B cells counts compared to placebo.
Results from a long-term extension trial of the drug also supported NICE’s decision, alongside further data that “addressed the areas of uncertainty particularly around the effects of stopping treatment that were identified by [the agency’s] independent committee”.
Also commenting on the recommendation, 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.”
