Pharming secures UK approval for Joenja in children aged 4–11
Key points
- Pharming Group (NASDAQ:PHAR) received UK approval to expand Joenja treatment to children aged 4–11 with activated PI3K delta syndrome (APDS).
- The approval covers children weighing at least 27 kg, with twice-daily doses of 40 mg or 50 mg.
- Pharming expects the expanded treatment indication to become available in February 2027 and plans to work with the NHS to support patient access.
Pharming Group (NASDAQ:PHAR) received UK regulatory approval to expand Joenja treatment to children aged 4–11 with APDS who weigh at least 27 kg, adding younger patients to its existing treatment population.
The approval expands the existing UK authorization for eligible adults and adolescents aged 12 and older, following the regulator's approval of 40 mg and 50 mg twice-daily dosing for younger children.
The Medicines and Healthcare products Regulatory Agency (MHRA) based its decision on Pharming's pediatric clinical programme, additional clinical pharmacology assessments and safety data, which showed that leniolisib was generally well tolerated in patients aged four years and above.
Pharming expects Joenja to become available to newly eligible UK patients in February 2027 and plans to work with the National Health Service (NHS) to support access.
Pharming develops and commercializes medicines for rare immune and genetic diseases, with Joenja serving as its targeted treatment for activated PI3K delta syndrome, a rare inherited condition affecting immune system development and function.
The company is expanding Joenja's availability across age groups and international markets, with existing approvals covering eligible patients in the United States, United Kingdom, European Union, Canada, Australia, Israel and South Korea, alongside a broader pediatric approval in Japan.
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Editorial verification notes
- The approval was announced on November 6, 2026.
- The regulator is the UK's Medicines and Healthcare products Regulatory Agency.
- Eligible children must be aged 4–11 and weigh at least 27 kg.
- Approved doses are 40 mg and 50 mg twice daily.
- Existing UK authorization covered eligible adults and adolescents aged 12 and older.
- Expected availability for the expanded indication is February 2027.
- The company intends to work with the NHS to facilitate access.
- Leniolisib was generally well tolerated in patients aged four years and above.
- The approval does not mean every child with APDS is eligible; the minimum weight requirement applies.
- The approval is a regulatory development, not evidence of revenue already generated from the newly eligible population.
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