- Approval extends existing U.K. indication for Joenja to eligible children aged 4 to 11 who weigh at least 27 kg
- Joenja is expected to become available to patients covered by the expanded indication in February 2027
- Marks another step in Pharming’s strategy to expand Joenja across age groups and international markets
Leiden, the Netherlands, November 6, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved an extension of the marketing authorization of Joenja®, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to include children aged 4 to 11 years weighing at least 27kg with activated PI3K delta syndrome (APDS), a rare primary immunodeficiency.
The approval includes 40 mg and 50 mg twice-daily dosing for eligible pediatric patients in this weight group, making Joenja the first treatment approved in the U.K. specifically for children with APDS.
The approval also expands the existing U.K. indication for Joenja, which was previously authorized for eligible adults and adolescents aged 12 years and older. Joenja is expected to become available to patients covered by the expanded indication in February. Pharming will work closely with the NHS and relevant stakeholders to support timely access for eligible patients.
"This approval is the latest step in our continued expansion of Joenja across age groups and geographies, giving eligible children in the U.K. aged 4 to 11 with APDS an approved treatment option," said Leverne Marsh, interim Co-CEO and Chief Commercial Officer of Pharming. "We will work closely with the NHS to support timely access for younger patients."
“The extension of the marketing authorization for leniolisib to children as young as four years old represents a significant step forward for children and families living with APDS. For families facing the devastating diagnosis of APDS, this extension provides an important new option: access to a targeted treatment supported by clinical data, without having to wait until their child reaches an older age,” said Dr Austen Worth, Pediatric Immunology Consultant at Great Ormond Street Hospital (GOSH) for Children and Associate Professor in Molecular Immunology at University College London, U.K.
The MHRA decision was supported by data from Pharming’s pediatric clinical program, conducted as part of the MHRA-agreed Pediatric Investigation Plan (PIP) for leniolisib, together with additional clinical pharmacology and safety assessments supporting the approved dosing regimen. Leniolisib was generally well-tolerated in patients of 4 years and above, supporting a positive benefit: risk profile in this age group.
Joenja® is a registered trademark owned by Pharming Group N.V. or its affiliates.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.8
About Joenja (leniolisib)
Joenja (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adults and adolescents 12 years of age and older in the U.S., U.K., Australia, Israel, the EU, Canada, and South Korea; in children 4 to 11 years of age who weigh at least 27 kg in the U.S., and U.K. and for patients 4 years of age and older in Japan. Joenja inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase III clinical trial demonstrated statistically significant improvement in the coprimary endpoints, reflecting a favorable impact on the immune dysregulation and deficiency seen in these patients, and open label extension data has supported the safety and tolerability of long-term leniolisib administration.9,10
Leniolisib is currently under regulatory review for the treatment of APDS in several other countries. Leniolisib is also being evaluated in two Phase II clinical trials in primary immunodeficiencies (PIDs) with immune dysregulation. The safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS.
About Pharming
Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biotechnology company that develops and commercializes innovative medicines for people living with rare immune and genetic diseases.
We combine specialized scientific, medical, regulatory and commercial expertise to advance a focused portfolio of approved medicines and development programs that address significant unmet medical needs. Guided by insights from patients and the wider rare disease community, we are dedicated to delivering innovative therapies for some of the most challenging rare diseases.
Pharming is headquartered in Leiden, the Netherlands, with operations in the United States and Europe.
For more information, visit www.pharming.com and find us on LinkedIn.
References
- Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.
- Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
- Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
- Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
- Maccari ME, et al. Front Immunol. 2018;9:543.
- Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.
- Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
- Vanselow S, et al. Frontiers in Immunology. 2023;14:1208567.
- Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
For further public information, contact:
Pharming
Michael Levitan, VP Investor Relations & Capital Markets
T: +1 (908) 705 1696
E: investor@pharming.com
Saskia Mehring, Head of Corporate Communications
T: +31 6 28 32 60 41
E: media.relations@pharming.com
Media Relations
Julia Deutsch (Lyra Strategic Advisory on behalf of Pharming)
E: JDeutsch@lyraadvisory.com
Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)
T: +31 6 53 81 64 27
Forward-looking Statements
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