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

Rhea-AI Summary

Pharming (EURONEXT:PHARM) announced a positive CHMP opinion recommending marketing authorization for Joenja (leniolisib) for treatment of APDS in patients aged 12 and older. A final European Commission decision is expected in Q2 2026, roughly two months after March 27, 2026.

The recommendation is based on a randomized Phase II/III trial (31 patients) showing statistically significant improvements in immune dysregulation and immunodeficiency, and on long-term open-label data from 37 patients with median three-year exposure. Joenja is already approved in the United States, United Kingdom, and Japan.

Positive

• CHMP positive opinion recommending EU marketing authorization
• First potential EU-approved treatment for APDS
• Phase II/III trial showed statistically significant clinical benefits in 31 patients
• Long-term safety/efficacy data: 37 patients, median three years exposure
• Existing approvals in United States, United Kingdom, and Japan

Negative

• Pivotal randomized trial enrolled only 31 patients
• Application seeks centralized authorization under exceptional circumstances
• Long-term data derived from an open-label extension without placebo control

Key Figures

Patient age threshold: 12 years and older Phase II/III sample size: 31 patients Extension trial patients: 37 patients +5 more

8 metrics

Patient age threshold 12 years and older Indicated APDS population in EU, US and UK approvals

Phase II/III sample size 31 patients Multinational triple-blind placebo-controlled randomized trial in APDS

Extension trial patients 37 patients Long-term open-label extension with leniolisib in APDS

Extension median duration 3 years Median leniolisib treatment in open-label extension study

Final EC decision timing Q2 2026 Expected European Commission decision on Joenja marketing authorization

EU Member States covered 27 Centralized authorization coverage plus Norway, Iceland, Liechtenstein

EC decision window 2 months Approximate time from CHMP opinion to EC decision

Publication date March 27, 2026 Date of positive CHMP opinion announcement

Market Reality Check

normal vol

Market Pulse Summary

This announcement centers on a positive CHMP opinion for Joenja in APDS, supported by a Phase II/III...

Analysis

This announcement centers on a positive CHMP opinion for Joenja in APDS, supported by a Phase II/III trial in 31 patients and an extension study with 37 patients treated for a median of 3 years. It highlights potential EU-wide coverage across 27 Member States plus associated countries. Investors may watch for the final European Commission decision expected in Q2 2026 and any further data on long-term safety and real-world use.

Key Terms

activated phosphoinositide 3-kinase delta (pi3kδ) syndrome, primary immunodeficiency, committee for medicinal products for human use, european medicines agency, +4 more

8 terms

activated phosphoinositide 3-kinase delta (pi3kδ) syndrome medical

"first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)"

Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome is a rare genetic immune disorder in which a specific signaling protein (PI3Kδ) is abnormally active, causing recurring infections, immune system overreaction, and increased risk of lymph node and organ enlargement. Investors care because the condition creates a defined patient population and clear biological target for drugs or genetic therapies; clinical trial results, regulatory decisions, or new treatments can materially affect companies developing therapies and their market value.

primary immunodeficiency medical

"activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult"

A group of inherited disorders in which parts of the immune system are missing or don’t work properly, leaving people unusually prone to infections, autoimmune complications, or certain cancers. Investors watch these conditions because they create steady, long‑term demand for diagnostics and specialized treatments (including replacement therapies and gene therapies); successful clinical trials or approvals can rapidly change a company’s revenue outlook and valuation, similar to discovering a cure for a chronic condition that many patients need.

committee for medicinal products for human use regulatory

"the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency"

The Committee for Medicinal Products for Human Use is the expert scientific panel within the European medicines regulator that assesses whether medicines for people are safe, effective and of acceptable quality, and issues formal opinions used in the drug-approval process. Its assessments act like a gatekeeper or safety inspector for entering the European market, so the committee’s opinion can materially affect a drug’s commercial prospects, regulatory risk and a company’s stock valuation.

european medicines agency regulatory

"Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)"

The European Medicines Agency is the central drug regulator that evaluates and authorizes medicines for use across the European Union and related countries, similar to a referee or safety inspector who checks that a medicine is safe and effective before it can be sold. Its decisions matter to investors because approvals, rejections, or safety warnings directly affect a drug maker’s ability to sell products, generate revenue, and face legal or reputational risks, which in turn influence stock value.

marketing authorization regulatory

"opinion recommending marketing authorization for Joenja"

An official government approval that allows a drug, vaccine, or medical device to be sold and promoted in a specific country or region. Think of it as a safety and effectiveness passport issued after regulators review the product’s tests and manufacturing; for investors, receiving this authorization typically unlocks sales, revenue potential, and lower regulatory risk, while delays or denials can substantially affect a company’s value and timeline.

triple-blind medical

"multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial"

Triple-blind describes a clinical trial design in which three parties—usually the patients, the clinicians giving the treatments, and the people who evaluate the results or analyze the data—are kept unaware of who received the active treatment or the control. Using an everyday analogy, it’s like a cooking contest where the chef, the judge, and the scorekeeper all don’t know which recipe used the secret ingredient. For investors, triple-blind trials reduce bias and increase confidence that reported safety and effectiveness findings are reliable, which affects a drug or device’s regulatory prospects and commercial value.

placebo-controlled medical

"multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

open-label extension medical

"long-term, open-label extension clinical trial in which 37 patients received leniolisib"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

AI-generated analysis. Not financial advice.

• If approved, Joenja® (leniolisib) would become the first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) 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 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 leniolisib
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 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.
  
Forward-looking Statements
This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2024 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information.

Inside Information
This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

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.

For further public information, contact:
Investor Relations
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
E: investor@pharming.com

Media Relations
Global: Saskia Mehring, Corporate Communications Manager
T: +31 6 28 32 60 41
E: media.relations@pharming.com

U.S.: Ethan Metelenis (Precision AQ on behalf of Pharming)
T: +1 (917) 882-9038

Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)
T: +31 6 53 81 64 27

Attachment

• FINAL_Pharming Group receives positive CHMP opinion for Joenja_EN_27Mar2026

FAQ

What did Pharming announce on March 27, 2026 about Joenja (PHARM)?

Pharming received a positive CHMP opinion recommending EU marketing authorization for Joenja. According to the company, a European Commission decision is expected in Q2 2026, about two months after the CHMP opinion.

Would Joenja (PHARM) be the first EU-approved treatment for APDS?

Yes — if authorized, Joenja would be the first approved APDS treatment in the EU. According to the company, the centralized authorization would cover all 27 EU member states plus Norway, Iceland, and Liechtenstein.

What clinical evidence supported the CHMP positive opinion for Joenja (PHARM)?

The opinion relied on a randomized Phase II/III trial showing statistically significant benefits in 31 patients. According to the company, additional long-term data came from 37 patients in an open-label extension with median three-year exposure.

When should investors expect the European Commission decision on Joenja (PHARM)?

A final European Commission decision is expected in Q2 2026, roughly two months after the March 27, 2026 CHMP opinion. According to the company, this timeframe targets a decision under exceptional circumstances.

Is Joenja (leniolisib) approved anywhere else besides Europe for APDS?

Yes — Joenja is already approved and marketed in the United States and the United Kingdom, and the company reported a recent approval in Japan earlier the same week as the CHMP opinion.