GlobeNewswire·Nov 6, 2026, 1:00 AM ET

MHRA approves Pharming’s Joenja® (leniolisib) as first treatment specifically for children with APDS in the U.K.

The U.K. MHRA approved an extension of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with activated PI3K delta syndrome (APDS).
This approval makes Joenja the first treatment in the U.K. specifically authorized for pediatric patients with APDS.
The approved dosing regimen for eligible children includes 40 mg or 50 mg administered twice daily.
Joenja is expected to become available to covered patients under the expanded indication in February 2027.
The decision supports Pharming's strategy to expand the drug across different age groups & international markets.
Clinical data from the Pediatric Investigation Plan demonstrated that leniolisib was generally well-tolerated in patients aged 4 years & above.
APDS is a rare primary immunodeficiency characterized by severe recurrent infections, lymphoproliferation, autoimmunity, & enteropathy.
Joenja is currently the only targeted treatment approved for APDS in multiple regions, including the U.S., EU, Australia, Israel, Canada, South Korea, & Japan.