- Joenja® (leniolisib) is first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients aged 12 years and older
- First European launch expected in Germany in Q3 2026
- Approval adds to Joenja® approvals in the United States, United Kingdom, Japan, Australia and Israel, supporting Pharming’s global expansion strategy
Leiden, the Netherlands, May 22, 2026: Pharming Group (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) today announced that the European Commission (EC) has granted marketing authorization for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older. Joenja is the first and only approved treatment for APDS in the European Union (EU). The first European launch is expected in Germany in Q3 2026, with additional launches anticipated pending completion of national reimbursement negotiations.
Leverne Marsh, Chief Commercial Officer of Pharming, commented:
“APDS is a progressive disease that can have a profound, lifelong impact on those living with the condition and their families, significantly affecting quality of life and underscoring the need for targeted treatment options. Today’s approval marks an important milestone for the European APDS community, making Joenja the first therapy in Europe specifically indicated to treat this disease.
This milestone reinforces our commitment to tackling rare disease challenges where new options are urgently needed. Joenja has the potential to meaningfully change how this complex condition is managed. We are proud to work with healthcare providers, patient communities and reimbursement authorities to support timely availability for eligible individuals across Europe.”
The EC approval is based on results from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, evaluating leniolisib in 31 patients with APDS aged 12 years and older. The study demonstrated statistically significant improvements in markers of immune dysregulation and immunodeficiency. The application also included long-term, open-label extension data from 37 patients who received leniolisib for a median of three years.
Prof. Ulrich Baumann, MD, Managing Senior Physician, Department of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Germany commented:
“The available clinical data on leniolisib consistently demonstrated a significant improvement in key parameters of immune dysregulation in patients with APDS. Of particular note is its targeted approach, which directly targets the underlying pathophysiology. Against the background of previously limited therapeutic options, this approval represents an important advancement in the management of this patient population.”
The approval is valid across all 27 EU Member States, as well as Norway, Iceland and Liechtenstein. Joenja is currently marketed in the United States and the United Kingdom for patients aged 12 years and older with APDS.
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 dysregulation1,2,3 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 treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in the U.S., U.K., Australia, Israel and the European Union in adult and pediatric patients 12 years of age and older and in Japan for patients 4 years of age and older. 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 Canada and 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 Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations.
For more information, visit www.pharming.com and find us on LinkedIn.
Forward-looking Statements
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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:
Investor Relations
Michael Levitan, VP Investor Relations and Capital Markets
T: +1 (908) 705 1696
E: investor@pharming.com
Media Relations
Global: Saskia Mehring, Head of Corporate Communications
T: +31 6 28 32 60 41
E: media.relations@pharming.com
U.S.: Christina Skrivan (Precision AQ on behalf of Pharming)
T: +1 (636)-352-7883
Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)
T: +31 6 53 81 64 27
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