European Commission grants marketing authorization to Pharming’s Joenja® (leniolisib) – the first approved treatment for APDS in the European Union

Rhea-AI Summary

Pharming (PHARM) received European Commission marketing authorization for Joenja (leniolisib), the first and only approved treatment in the EU for activated PI3K delta syndrome (APDS) in patients aged 12 and older.

First EU launch is expected in Germany in Q3 2026, with further roll-out after national reimbursement decisions. The approval, based on a Phase II/III trial and long-term extension data, is valid in all 27 EU states plus Norway, Iceland and Liechtenstein, and supports Pharming’s rare disease expansion alongside existing approvals in the US, UK, Japan, Australia and Israel.

AI-generated analysis. Not financial advice.

Positive

• EU marketing authorization for Joenja as first APDS treatment for patients 12+
• Approval covers 27 EU Member States plus Norway, Iceland and Liechtenstein
• First European launch planned in Germany in Q3 2026
• Supports global expansion with prior Joenja approvals in US, UK, Japan, Australia, Israel
• Phase II/III trial showed statistically significant improvements in immune markers
• Long-term extension data include 37 patients treated for median of three years

Negative

• None.

Key Figures

Patient age threshold: 12 years and older Phase II/III trial size: 31 patients Extension cohort size: 37 patients +3 more

6 metrics

Patient age threshold 12 years and older Indicated APDS population in EU approval

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

Extension cohort size 37 patients Open-label extension data included in EC application

Extension median duration three years Median leniolisib exposure in open-label extension

EU member states covered 27 EU Member States Territorial scope of approval plus Norway, Iceland, Liechtenstein

First EU launch timing Q3 2026 Planned initial Joenja launch in Germany

Market Reality Check

normal vol

Market Pulse Summary

This announcement highlights the European Commission’s marketing authorization for Joenja, the first...

Analysis

This announcement highlights the European Commission’s marketing authorization for Joenja, the first approved treatment for APDS in the EU, covering patients aged 12 and older. The decision applies across 27 EU Member States plus Norway, Iceland and Liechtenstein and is supported by a Phase II/III trial in 31 patients and an open-label extension in 37 patients with a median of three years exposure. Investors may track the planned Q3 2026 German launch, national reimbursement negotiations, and real-world uptake as key metrics.

Key Terms

marketing authorization, activated phosphoinositide 3-kinase delta (pi3kδ) syndrome (apds), primary immunodeficiency, phosphoinositide 3-kinase (pi3k) delta inhibitor, +4 more

8 terms

marketing authorization regulatory

"European Commission (EC) has granted 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.

activated phosphoinositide 3-kinase delta (pi3kδ) syndrome (apds) medical

"for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare"

A rare, inherited immune disorder caused by genetic changes that leave a key immune “on” switch — the PI3Kδ enzyme — stuck in a high-activity state, which weakens infection control and can cause swollen lymph nodes, autoimmune problems and higher cancer risk. For investors, APDS matters because it defines a clearly identifiable patient group with an urgent unmet need and a specific biological target, shaping clinical trial design, regulatory pathways and the commercial opportunity for therapies aimed at correcting that overactive pathway.

primary immunodeficiency medical

"syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric"

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.

phosphoinositide 3-kinase (pi3k) delta inhibitor medical

"an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment"

A phosphoinositide 3-kinase (PI3K) delta inhibitor is a type of drug that blocks a specific enzyme found mainly in immune cells, slowing signals that can drive uncontrolled cell growth or inflammation. Investors care because drugs in this class can treat certain cancers and autoimmune disorders; success in clinical trials or regulatory approval can meaningfully affect a company’s revenue outlook, while safety or efficacy setbacks can reduce a drug’s market potential.

triple-blind medical

"a 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

"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.

randomized phase ii/iii clinical trial medical

"placebo-controlled, randomized Phase II/III clinical trial, evaluating leniolisib"

A randomized phase II/III clinical trial combines mid-stage and late-stage testing of a new drug or treatment by randomly assigning patients to the experimental therapy or a comparison group to measure safety and how well it works in a larger population. Investors watch these trials because their outcomes heavily influence whether regulators will approve the product and how large its market could be—positive results can validate commercial potential, while failures raise uncertainty and value risk, like a final test drive before mass production.

open-label extension medical

"included long-term, open-label extension data from 37 patients who received"

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.

• 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 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 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
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 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, 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 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

Attachment

• EC grants marketing authorization to Pharmings Joenja_EN_22MAY2026

FAQ

What did Pharming (PHARM) announce about Joenja approval in the European Union on May 22, 2026?

Pharming announced that the European Commission granted marketing authorization for Joenja (leniolisib) to treat APDS in patients aged 12 and older. According to Pharming, Joenja is now the first and only approved APDS treatment across the European Union and associated countries.

For which patients is Joenja (leniolisib) now approved in the EU under Pharming’s marketing authorization (PHARM)?

Joenja is approved for adults and pediatric patients aged 12 years and older with activated PI3K delta syndrome (APDS). According to Pharming, it is an oral, selective PI3K delta inhibitor targeting this rare primary immunodeficiency in eligible patients across the EU and certain EEA countries.

When and where will Pharming (PHARM) first launch Joenja in Europe after EU approval?

The first European launch of Joenja is expected in Germany in Q3 2026. According to Pharming, additional country launches are anticipated following completion of national reimbursement negotiations, supporting broader access for eligible APDS patients across Europe over time.

What clinical data supported the European Commission’s approval of Joenja for APDS for Pharming (PHARM)?

Approval was supported by a multinational, triple-blind, placebo-controlled Phase II/III trial in 31 APDS patients aged 12 and older. According to Pharming, the study showed statistically significant improvements in immune dysregulation markers, supplemented by long-term extension data from 37 patients over a median three years.

Which European countries are covered by Pharming’s Joenja (PHARM) marketing authorization for APDS?

The marketing authorization is valid in all 27 European Union Member States plus Norway, Iceland and Liechtenstein. According to Pharming, this broad geographic coverage enables potential access to Joenja for eligible APDS patients across much of Europe, subject to national reimbursement processes.

How does Joenja’s EU approval fit into Pharming’s global strategy for APDS (PHARM)?

The EU approval advances Pharming’s global rare disease expansion strategy for Joenja. According to Pharming, Joenja already has approvals in the United States, United Kingdom, Japan, Australia and Israel, and the new European authorization further broadens its international treatment footprint for APDS.