Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (NASDAQ:PHAR) revealed on Wednesday that it has received a positive final recommendation from the National Institute for Health and Care Excellence (NICE) for Joenja (leniolisib) as a treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
The recommendation covers adult and paediatric patients aged 12 and over within the National Health Service (NHS) in England and Wales, making Joenja the first and only APDS-specific treatment to be reimbursed by the NHS.
NICE based its decision on data from a Phase III trial demonstrating significant improvements in immune function and disease control in patients treated with leniolisib, a selective oral PI3Kδ inhibitor.
These outcomes were sustained in an open-label extension study, reinforcing leniolisib's long-term clinical benefit for this rare and progressive immunodeficiency.
The recommendation aligns with the UK Medicines and Healthcare products Regulatory Authority approval and is issued under NICE's Highly Specialised Technologies pathway.
Joenja is now reimbursed in England through the Innovative Medicines Fund, providing immediate access to eligible patients. In Wales, funding is expected within three months through NHS specialist centres.
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