Pharming: Joenja approved in the United Kingdom for children aged 4 to 11
The British Medicines and Healthcare products Regulatory Agency (MHRA) has extended the marketing authorisation of Joenja (leniolisib) to children aged 4 to 11 weighing at least 27 kg and suffering from APDS, a rare primary immunodeficiency.
Pharming, which announced this decision in a press release on 6 November 2026, states that the treatment should be accessible to these young patients in February 2027.
A paediatric indication added to those for adults and adolescents
The authorisation covers doses of 40 mg and 50 mg administered twice daily for children in this weight category. According to Pharming, Joenja thus becomes the first treatment authorised in the United Kingdom specifically for children with APDS, whereas it had until now only been authorised from the age of 12.
The group presents this decision as a new step in its strategy of extending Joenja to different age groups and to new international markets.
Leverne Marsh, interim co-chief executive officer and chief commercial officer, stated: "This approval is the latest step in our continued expansion of Joenja across age groups and geographies".
The medicine is now authorised for children aged 4 to 11 weighing at least 27 kg in the United States and the United Kingdom, and from age 4 in Japan. It is authorised from age 12 in the European Union, Canada, Australia, Israel and South Korea, and remains under regulatory review in several other countries.
A decision supported by the paediatric investigation plan
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The MHRA relied on data from Pharming's paediatric clinical programme, conducted under the paediatric investigation plan agreed with the agency, supplemented by clinical pharmacology and safety assessments. According to the press release, leniolisib was "generally well tolerated" in patients aged 4 years and older.
Pharming states that it will work with the NHS and relevant stakeholders to facilitate access for eligible patients. Dr Austen Worth, paediatric immunologist at Great Ormond Street Hospital, believes this extension gives families access to targeted treatment "without having to wait for their child to reach an older age".
APDS, first characterised in 2013, affects approximately 1 to 2 people per million worldwide. According to the document, patients experience a median diagnostic delay of seven years, whereas the disease is progressive and can lead to permanent lung damage and lymphomas.
SectorPharmacie et biotechnologies›Biotechnologies
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Context
Period
Period: T3 2026
Key reported figures
Revenue: 117,6 millions de dollars
Quarterly revenue: 117,6 millions de dollars
Revenue growth: 21 %
Guidance from the release
Tightened 2026 total revenue guidance to between US$380 million and US$390 million from prior US$375 million to US$395 million
Risks mentioned
Joenja revenue growth was partly driven by temporary benefit from ordering patterns in other international markets
RUCONEST third quarter revenue includes an early seasonal increase in channel inventory at a single U.S. specialty pharmacy customer, following significant drawdowns earlier in the year
Napazimone (KL1333): enrollment in the pivotal FALCON trial now expected to complete in the first half of 2027 (about 145 of 180 subjects by end-2026), with topline results approximately one year after completion of enrollment
Opportunities identified
RUCONEST stabilization and continued recovery of demand in the US (third quarter revenue of about US$93.8 million, +14% year-on-year and +30% versus the second quarter of 2026)
Joenja strong growth driven by launches in Germany, Japan and continued penetration in the U.K.
Substantial growth potential for Joenja with estimated CVID with immune dysregulation prevalence of 39 patients per million vs 1.5 for APDS
Outlook / guidance
Expected revenue: 385 millions de dollars
Management commentary: Tightened 2026 total revenue guidance to between US$380 million and US$390 million (1% to 4% growth) from prior US$375 million to US$395 million (0% to 5% growth)
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