Pharming Group N.V.: Pharming Group receives positive CHMP opinion for Joenja (leniolisib) for the treatment of APDS in adult and pediatric patients 12 years and older

• If approved, Joenja® (leniolisib) would become the first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS), a rare primary immunodeficiency
• Decision based on Phase II/III clinical data demonstrating statistically significant impact on measures of immune dysregulation and immunodeficiency
• Final European Commission decision expected in Q2 2026
  
  

Leiden,the Netherlands, March 27, 2026, Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older.

A final decision by the European Commission on the marketing authorization for Joenja under exceptional circumstances is expected within approximately two months. If approved, Joenja would become the first approved treatment for APDS in the European Union. The centralized marketing authorization would be valid in all 27 European Union Member States, as well as Norway, Iceland and Liechtenstein.

Fabrice Chouraqui, Chief Executive Officer of Pharming, commented:
"For patients living with APDS, there remains a significant unmet medical need. By targeting the underlying cause of the disease, Joenja could mark a step-change in APDS care in Europe. Today's positive CHMP opinion, together with the approval in Japan earlier this week, reflects the strength of the clinical data and the dedication of the patients, families, and physicians who participated in the clinical studies. We look forward to the European Commission's final decision and to working with relevant authorities across Europe to support patient access."

The positive CHMP opinion is based on results from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older and demonstrated a statistically significant impact on immune dysregulation and immunodeficiency. Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 37 patients received leniolisib for a median of three years.

Virgil Dalm, MD, PhD, Head of Division of Allergy & Clinical Immunology of the Department of Internal Medicine and Principal Investigator, Erasmus University Medical Center, Rotterdam, the Netherlands, commented:
"The clinical program for leniolisib has shown consistent, meaningful improvements across markers of both immune dysregulation and immune deficiency in patients with APDS. Taken together with a favorable safety profile, these results suggest Joenja could meaningfully change the clinical outlook for people living with this rare, complex and progressive inborn error of immunity. As a physician and investigator in the clinical program, I know how important it is to have treatment options that address the underlying cause of disease and have the potential to make a meaningful difference for patients and their families. I am proud to see the dedication of investigators and the patients who participated in the studies contribute to this milestone, and I look forward to the European Commission's decision and the potential for patients across Europe to access this novel targeted therapy."

Joenja is approved and marketed in the United States and the United Kingdom for patients aged 12 years and older with APDS.

About Activated Phosphoinositide 3-Kinase d 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 PI3Kd (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation¹²³ APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.⁴⁵ 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.⁴⁷ A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.⁸

About leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor approved as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS) in the U.S., U.K., Australia and Israel in adult and pediatric patients 12 years of age and older and in Japan for patients 4 years of age and older. Leniolisib 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 are developing and commercializing a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with a significant proportion of its employees based in the U.S.

For more information, visit www.pharming.com and find us on LinkedIn.
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References

1. Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
8. Vanselow S, et al. Frontiers in Immunology. 2023;14:1208567.
9. Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
10. Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.

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