Leiden, the Netherlands, May 7, 2026
Pharming Group N.V. announced a major scientific presence at the 2026 Annual Meeting of the Clinical Immunology Society (CIS) in New Orleans, presenting new clinical findings supporting the expanding role of leniolisib in the treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) and broader primary immunodeficiencies characterized by immune dysregulation. Across multiple poster presentations, the company and collaborating investigators showcased long-term pediatric safety and efficacy data, expanded access experiences in Common Variable Immunodeficiency (CVID) and CVID-like disorders, and new insights into disease burden and symptom improvement in pediatric APDS patients. The data further strengthen Pharming’s position in the rare immunology market while reinforcing the growing clinical relevance of targeted PI3Kδ inhibition for immune-mediated disorders with high unmet medical need.
Long-Term Pediatric APDS Data Strengthens Leniolisib Position
One of the most significant presentations at CIS 2026 focused on interim safety and efficacy outcomes from an ongoing open-label long-term extension study evaluating leniolisib in pediatric APDS patients aged 4 to 11 years. The study demonstrated continued therapeutic benefit alongside a favorable tolerability profile, adding to previously established Phase III clinical evidence supporting leniolisib as the first and only approved targeted therapy for APDS in multiple global markets, including the United States, United Kingdom, Australia, Israel, and Japan.
APDS is an ultra-rare inherited immunodeficiency caused by variants in the PIK3CD or PIK3R1 genes that trigger hyperactivation of the PI3Kδ signaling pathway, leading to recurrent infections, immune dysregulation, autoimmunity, lymphoproliferation, and elevated lymphoma risk. Because symptoms overlap with several other immune disorders, APDS patients often face years of delayed diagnosis, increasing the risk of irreversible organ damage and disease progression. The pediatric data presented at CIS 2026 further validate leniolisib’s ability to improve disease management while addressing one of the most severe underlying molecular drivers of APDS.
Expanded Clinical Experience Supports Broader Immune Dysregulation Strategy
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Beyond APDS, Pharming also presented important clinical observations from expanded access use of leniolisib in patients suffering from CVID and CVID-like disorders, suggesting the drug’s potential applicability across a broader range of primary immunodeficiencies involving immune dysregulation. These findings support the company’s ongoing Phase II clinical trials evaluating leniolisib in additional immune disorders beyond APDS. Researchers highlighted meaningful clinical improvements and symptom management outcomes that may open future regulatory and commercial opportunities in rare immunology markets where treatment options remain limited.
Additional presentations explored symptom changes in pediatric APDS patients receiving leniolisib, caregiver burden associated with managing APDS, and systematic reviews involving sirolimus and immunoglobulin replacement therapy usage patterns. Collectively, the presentations reinforce growing physician interest in targeted immunomodulatory therapies capable of modifying disease progression rather than merely managing symptoms. Pharming’s expanding leniolisib development strategy could significantly broaden the therapeutic landscape for patients with difficult-to-diagnose immune disorders driven by PI3K pathway dysfunction.
CIS 2026 Presentations Reinforce Pharming’s Rare Disease Leadership
Pharming’s extensive participation at CIS 2026 reflects the company’s aggressive effort to establish itself as a global leader in rare immunology and precision medicine therapies. Chief Medical Officer Anurag Relan emphasized that the newly presented data continue advancing scientific understanding of APDS and related primary immunodeficiencies while supporting ongoing clinical development initiatives. With two active Phase II studies currently underway and additional regulatory reviews ongoing internationally, Pharming is positioning leniolisib as a foundational targeted therapy platform within rare immune diseases.
The company’s strategy aligns with growing industry demand for precision therapeutics capable of directly targeting molecular disease drivers rather than relying solely on broad immunosuppression approaches. As awareness of APDS continues increasing globally through improved genetic testing and physician education, the commercial opportunity for targeted PI3Kδ inhibitors may expand significantly in coming years. The latest CIS data further strengthen confidence in leniolisib’s long-term safety profile, pediatric utility, and broader clinical potential across multiple immune dysregulation disorders.
Source: Pharming Group press release
