MHRA approves Pharming’s Joenja® (leniolisib) as first treatment specifically for children with APDS in the U.K.

The expanded authorization adds eligible younger children to Joenja’s existing U.K. treatment population.

Rhea-AI Summary

Pharming (Nasdaq: PHAR) received U.K. approval to expand Joenja treatment to children aged 4 to 11 with APDS. The expanded indication covers children weighing at least 27 kg and includes 40 mg and 50 mg twice-daily dosing. APDS, or activated PI3K delta syndrome, is a rare inherited immune disorder. Joenja was previously authorized in the U.K. for eligible adults and adolescents aged 12 years and older.

Pharming expects availability for newly eligible patients in February 2027 and plans to work with the NHS to support access. The MHRA decision was supported by the pediatric clinical program and additional clinical pharmacology and safety assessments supporting the dosing regimen. Leniolisib, Joenja’s active ingredient, was generally well-tolerated in patients aged 4 years and above.

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• Moderate pointJoenja’s U.K. approval expands treatment to children aged 4 to 11 weighing at least 27 kg.
• Minor point. Forward-looking: it has not happened yet and may not happen.Expanded-indication availability is expected in February 2027, with Pharming planning NHS access support.
• Minor pointLeniolisib was generally well-tolerated in patients aged 4 years and above.

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• None.

Key Figures

Eligible pediatric group: 4–11 years; at least 27 kg Approved dosing: 40 mg and 50 mg twice daily Expected availability: February 2027

Eligible pediatric group

4–11 years; at least 27 kg

U.K. expanded indication

Approved dosing

40 mg and 50 mg twice daily

Eligible pediatric patients in this weight group

Expected availability

February 2027

Patients covered by the expanded indication

Historical Context

1 past event · Latest: Oct 27

1 event

1. Oct 27

   Earnings report

   24h Move

   -0.3%

   Reported U.S. shipments of newly approved pediatric Joenja doses had begun in October.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, marketing authorization, pediatric investigation plan (PIP), clinical pharmacology, +1 more

5 terms

phosphoinositide 3-kinase delta (PI3Kδ) inhibitor medical

"an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor"

A phosphoinositide 3-kinase delta (PI3Kδ) inhibitor is a drug or compound that selectively blocks the activity of the delta isoform of the phosphoinositide 3-kinase enzyme family. By inhibiting PI3Kδ, which is mainly active in white blood cells, these agents interrupt intracellular signaling pathways that control immune cell activation, proliferation and survival—effects exploited in treating certain blood cancers and immune-mediated diseases. The term denotes selectivity for the delta isoform (distinct from PI3K alpha, beta or gamma), so properties and side-effect profiles differ from nonselective PI3K inhibitors.

marketing authorization regulatory

"an extension of the marketing authorization of 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.

pediatric investigation plan (PIP) regulatory

"the MHRA-agreed Pediatric Investigation Plan (PIP) for leniolisib"

A pediatric investigation plan (PIP) is a regulator-approved blueprint that outlines how a medicine or medical product will be tested, dosed and studied in children. Like a construction plan for adapting a building to a new age group, it sets required studies, timing and safety measures so regulators can judge use in pediatric patients. For investors, a PIP affects development cost, timeline and approval chances, and can influence future sales and exclusivity.

clinical pharmacology medical

"additional clinical pharmacology and safety assessments"

Clinical pharmacology is the study of how medicines behave in humans — how they are absorbed, distributed, metabolized and eliminated, and what effects and side effects they produce at different doses. For investors, it matters because these studies determine safe and effective dosing, identify risks, shape clinical trial outcomes and regulatory decisions, and therefore influence a drug’s chances of approval, market size and commercial value; think of it as the science that maps a drug’s real-world performance.

activated PI3K delta syndrome (APDS) medical

"with activated PI3K delta syndrome (APDS), a rare primary immunodeficiency"

A rare inherited primary immunodeficiency caused by gain‑of‑function mutations that make the PI3Kδ (phosphoinositide 3‑kinase delta) signaling pathway overactive; the overactivity impairs normal immune cell development and function and leads to recurrent infections, enlarged lymph nodes or spleen, increased risk of lymphoproliferation and autoimmunity. The condition is genetic—most commonly due to mutations in the PIK3CD or PIK3R1 genes—and clinical features and severity vary widely between individuals because different mutations and modifier factors produce different degrees of pathway activation.

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• Approval extends existing U.K. indication for Joenja to eligible children aged 4 to 11 who weigh at least 27 kg
• Joenja is expected to become available to patients covered by the expanded indication in February 2027
• Marks another step in Pharming’s strategy to expand Joenja across age groups and international markets
  
  

Leiden, the Netherlands, November 6, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved an extension of the marketing authorization of Joenja®, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to include children aged 4 to 11 years weighing at least 27kg with activated PI3K delta syndrome (APDS), a rare primary immunodeficiency.

The approval includes 40 mg and 50 mg twice-daily dosing for eligible pediatric patients in this weight group, making Joenja the first treatment approved in the U.K. specifically for children with APDS.

The approval also expands the existing U.K. indication for Joenja, which was previously authorized for eligible adults and adolescents aged 12 years and older. Joenja is expected to become available to patients covered by the expanded indication in February. Pharming will work closely with the NHS and relevant stakeholders to support timely access for eligible patients.

"This approval is the latest step in our continued expansion of Joenja across age groups and geographies, giving eligible children in the U.K. aged 4 to 11 with APDS an approved treatment option," said Leverne Marsh, interim Co-CEO and Chief Commercial Officer of Pharming. "We will work closely with the NHS to support timely access for younger patients."

“The extension of the marketing authorization for leniolisib to children as young as four years old represents a significant step forward for children and families living with APDS. For families facing the devastating diagnosis of APDS, this extension provides an important new option: access to a targeted treatment supported by clinical data, without having to wait until their child reaches an older age,” said Dr Austen Worth, Pediatric Immunology Consultant at Great Ormond Street Hospital (GOSH) for Children and Associate Professor in Molecular Immunology at University College London, U.K.

The MHRA decision was supported by data from Pharming’s pediatric clinical program, conducted as part of the MHRA-agreed Pediatric Investigation Plan (PIP) for leniolisib, together with additional clinical pharmacology and safety assessments supporting the approved dosing regimen. Leniolisib was generally well-tolerated in patients of 4 years and above, supporting a positive benefit: risk profile in this age group.

Joenja® is a registered trademark owned by Pharming Group N.V. or its affiliates.

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 targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adults and adolescents 12 years of age and older in the U.S., U.K., Australia, Israel, the EU, Canada, and South Korea; in children 4 to 11 years of age who weigh at least 27 kg in the U.S., and U.K. and for patients 4 years of age and older in Japan.  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 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
Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biotechnology company that develops and commercializes innovative medicines for people living with rare immune and genetic diseases.

We combine specialized scientific, medical, regulatory and commercial expertise to advance a focused portfolio of approved medicines and development programs that address significant unmet medical needs. Guided by insights from patients and the wider rare disease community, we are dedicated to delivering innovative therapies for some of the most challenging rare diseases.

Pharming is headquartered in Leiden, the Netherlands, with operations in the United States and Europe. 

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

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:

Pharming
Michael Levitan, VP Investor Relations & Capital Markets 
T: +1 (908) 705 1696 
E: investor@pharming.com 

Saskia Mehring, Head of Corporate Communications 
T: +31 6 28 32 60 41 
E: media.relations@pharming.com

Media Relations 

Julia Deutsch (Lyra Strategic Advisory on behalf of Pharming)
E: JDeutsch@lyraadvisory.com
Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming) 
T: +31 6 53 81 64 27 

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.

Attachment

• Pharming receives UK MHRA approval Joenja for children_EN_06NOV2026

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

Which children are eligible for Pharming’s expanded U.K. Joenja approval?

The expanded U.K. approval covers children with APDS aged 4 to 11 years who weigh at least 27 kg. It includes 40 mg and 50 mg twice-daily dosing and extends the existing authorization for eligible adults and adolescents aged 12 years and older.

When will Joenja become available to newly eligible children in the U.K.?

Pharming expects Joenja to become available to patients covered by the expanded indication in February 2027. The company plans to work with the NHS and relevant stakeholders to support timely access for eligible patients.