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news.The National Institute for Health and Care Excellence (NICE) has issued final guidance endorsing Pharming Group’s Joenja (leniolisib) for use and reimbursement in the National Health Service (NHS) in England and Wales.
The oral therapy is now accessible and funded in England via the Innovative Medicines Fund. Credit: Hal Gatewood on Unsplash.
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This recommendation facilitates treating the activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 years and above.
The oral therapy is now accessible and funded in England through the Innovative Medicines Fund and is expected to be funded in Wales within the coming three months.
The NICE endorsement is supported by the comprehensive data, including a Phase III clinical trial of leniolisib, which showed significant improvements in immune deficiency and dysregulation in APDS subjects against a placebo.
This trial also indicated sustained clinical benefits during extended treatment in the open-label extension study.
The positive NICE guidance is in line with the UK Medicines and Healthcare products Regulatory Authority (MHRA) approval.
It comes under the Highly Specialised Technologies (HST) pathway for rare and severe conditions.
APDS is stated to be a rare primary immunodeficiency that occurs due to mutations in the PIK3CD or PIK3R1 genes, essential for immune cell development and function.
Joenja claims to be the first and only targeted treatment for this condition, which gained approval in the UK, Australia, Israel and the US, for the adult and paediatric population aged 12 years and above.
Currently, the therapy is being reviewed in Canada, the European Economic Area, and other nations for APDS.
There are also plans to seek regulatory approval in Japan.
Additional Phase III trials in paediatric patients with APDS and Phase II trials in primary immunodeficiencies with immune dysregulation are underway.
Pharming Group CEO Fabrice Chouraqui said: “This positive recommendation for Joenja from NICE is a significant milestone for patients living with APDS in England and Wales.
“It underscores Pharming’s commitment to work closely with regulatory and reimbursement authorities, the medical immunology community, and patient associations to bring this first targeted treatment to patients who may benefit.”
