- Joenja® (leniolisib) receives positive recommendation from NICE, becoming the first and only treatment for APDS approved within the NHS.
- The approval is for adult and pediatric patients 12 years and older, ensuring immediate access in England.
- Phase III trials demonstrated significant improvements in immune deficiency and dysregulation.
Pharming Group N.V. (PHARM, Financial) has announced that the National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for Joenja® (leniolisib) in the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). This makes Joenja the first and only approved therapy for APDS available within the National Health Service (NHS) in England and Wales.
The recommendation is based on robust data from Phase III clinical trials, which demonstrated significant improvements in immune deficiency and dysregulation compared to placebo, with these benefits sustained during long-term treatment in an extension trial.
Joenja is recommended for both adult and pediatric patients aged 12 years and older, addressing a crucial gap in treatment for this rare immunodeficiency disorder. Immediate availability is assured in England through the Innovative Medicines Fund, while Welsh access will follow within three months via NHS specialist centers.
The decision by NICE under the Highly Specialised Technologies pathway is a significant milestone, aligning with the U.K. Medicines and Healthcare products Regulatory Authority (MHRA) approval. This endorsement could potentially accelerate similar market access decisions in other European countries, as Pharming continues to expand the availability of Joenja across global markets.
