Pharming announces U.S. FDA acceptance of sNDA resubmission for Joenja® (leniolisib) to treat children aged 4 to 11 years with APDS

Rhea-AI Summary

Pharming (PHARM) announced U.S. FDA acceptance of its resubmitted sNDA for Joenja (leniolisib) to treat children aged 4–11 years with APDS.

The filing seeks approval of 40 mg and 50 mg twice-daily dosing for patients ≥27 kg and has an FDA PDUFA target action date of October 24, 2026. If approved, Joenja would be the first U.S. treatment for children with APDS. The resubmission includes additional analytical data requested after a January 30, 2026 CRL and is supported by a Phase III pediatric study showing improvements in lymphadenopathy and naïve B cells, with only mild to moderate adverse events and no drug-related serious events.

Positive

• FDA acceptance of Joenja sNDA resubmission for children aged 4–11 years with APDS
• PDUFA target action date set for October 24, 2026, providing a clear review timeline
• Phase III pediatric study showed improvements in lymphadenopathy and naïve B cells over 12 weeks
• Treatment-emergent adverse events were mild to moderate, with no drug-related serious events reported
• All pediatric patients in the Phase III study completed the 12-week Joenja treatment period
• Planned separate sNDA for lower-dose Joenja in patients weighing under 27 kg in second half 2026

Negative

• Previous Complete Response Letter from FDA on January 30, 2026 highlights prior regulatory hurdles for Joenja
• Current sNDA dosing covers only pediatric patients weighing 27 kg or more, leaving lighter children pending another filing
• FDA decision on the pediatric sNDA remains uncertain until at least the October 24, 2026 PDUFA date

Market Context

This announcement details FDA acceptance of Pharming’s resubmitted sNDA for Joenja in children aged ...

Analysis

This announcement details FDA acceptance of Pharming’s resubmitted sNDA for Joenja in children aged 4–11 with APDS, with a PDUFA action date of October 24, 2026. The filing is backed by Phase III data over 12 weeks showing improvements in APDS hallmarks and only mild to moderate adverse events. Investors may track regulatory milestones after the prior CRL on January 30, 2026 and the planned sNDA for patients under 27 kg.

Key Figures

PDUFA target date: October 24, 2026 CRL date: January 30, 2026 Type A meeting: March 26, 2026 +5 more

8 metrics

PDUFA target date October 24, 2026 U.S. FDA action date for Joenja sNDA in children 4–11

CRL date January 30, 2026 Complete Response Letter for Joenja pediatric sNDA

Type A meeting March 26, 2026 Follow-up meeting with FDA after CRL

Pediatric age range 4–11 years Targeted APDS pediatric population for Joenja sNDA

Dosing strengths 40 mg and 50 mg BID Twice-daily dosing for patients ≥27 kg in resubmission

Weight threshold 27 kg Cutoff for current pediatric dosing sNDA; lower weight sNDA planned

Treatment duration 12 weeks Phase III study period with improvements in APDS hallmarks

Prior FDA approval age ≥12 years Existing U.S. approval for APDS since March 2023

Key Terms

snda, complete response letter, type a meeting, pdufa, +4 more

8 terms

snda regulatory

"Supplemental New Drug Application (sNDA) resubmission seeks approval"

A SNDA (Subordination, Non‑Disturbance and Attornment Agreement) is a legal pact among a property owner’s lender, the owner’s tenants, and sometimes the landlord that sets who keeps lease rights if the property is sold or a mortgage is enforced. Think of it as a rulebook that decides whether a tenant can stay and keep paying rent or must answer to a new owner after a foreclosure. For investors, an SNDA matters because it protects predictable rental income, clarifies who has priority on claims against a property, and therefore affects a property’s value and the security of related loans.

complete response letter regulatory

"Following the Complete Response Letter (CRL) received on January 30, 2026"

A complete response letter is an official communication from a drug or medical-device regulator, such as the U.S. Food and Drug Administration (FDA), telling a company that a marketing application cannot be approved in its current form and listing the specific deficiencies to be fixed. For investors it matters because it pauses or delays a product’s path to market—like a building inspector issuing a list of repairs before a certificate of occupancy—affecting revenue timing, costs and stock value.

type a meeting regulatory

"and a subsequent Type A meeting with the FDA on March 26, 2026"

A Type A meeting is an urgent, short-notice session requested between a company and a regulatory agency (for example, the FDA in the U.S.) to resolve critical issues that block a development program, such as a clinical hold or safety concern. Investors care because the outcome can immediately affect whether a clinical trial or approval process resumes, changing timelines, costs and the company’s near-term value — like calling an emergency mechanic when a car won’t start so a trip can continue.

pdufa regulatory

"The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date"

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

phase iii medical

"supported by positive data from the open-label, multinational, single-arm Phase III study"

A Phase III trial is the late-stage clinical study that tests whether a medical treatment works and is safe in a large group of patients, often comparing it to standard care. Think of it as a final dress rehearsal or full-scale road test before regulators decide on approval; positive or negative results strongly influence a drug maker’s chance to sell the treatment, future revenue, and investment risk.

lymphadenopathy medical

"which showed improvements over 12 weeks in two clinically relevant hallmarks of APDS, reduced lymphadenopathy"

Swollen or abnormal lymph nodes caused by infection, inflammation, immune reactions, or cancer; they are the body's drainage and surveillance hubs, like neighborhood security posts that enlarge when something is wrong. Investors care because lymphadenopathy reported in clinical trials or product safety data can signal an adverse effect, disease progression, or efficacy issue that may delay approvals, change market expectations, or affect a company’s valuation.

naïve b cells medical

"and increased naïve B cells, together indicating a correction of the underlying immune defect"

Naïve B cells are immune cells that have not yet encountered a specific germ or vaccine; they act like an untrained toolkit ready to be taught to recognize and fight new threats. For investors, their number and responsiveness matter because they influence how well vaccines, antibody drugs, or immune-based therapies can generate protective antibodies and long-term immunity, affecting clinical trial outcomes and product durability.

treatment emergent adverse events medical

"All treatment emergent adverse events were reported to be mild to moderate in nature"

Treatment emergent adverse events are any new or worsened medical problems that appear after a patient starts a drug or medical intervention during a clinical trial. Investors care because the number, severity, and frequency of these events influence safety profiles, regulatory approval chances, and market acceptance; think of them like unexpected problems that crop up after installing a software update—minor ones may be manageable, but serious or common issues can stall or derail the product.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• If approved, Joenja will be the first approved treatment in the U.S. for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency
• Supplemental New Drug Application (sNDA) resubmission seeks approval for 40 mg and 50 mg twice-daily dosing
• Separate sNDA for lower weight pediatric patients planned for second half 2026
  
  

Leiden, the Netherlands, June 4, 2026: Pharming Group (Euronext: PHARM; Nasdaq: PHAR) today announced that the U.S. Food and Drug Administration (FDA) has accepted its resubmitted supplemental New Drug Application (sNDA) seeking approval for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. Following the Complete Response Letter (CRL) received on January 30, 2026, and a subsequent Type A meeting with the FDA on March 26, 2026, the resubmission seeks approval of 40 mg and 50 mg twice-daily dosing for pediatric patients weighing 27 kg or more, who represent a meaningful proportion of the identified pediatric patient population. The resubmission includes additional data requested by the FDA on analytical methods used for production batch testing. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 24, 2026.

A separate sNDA seeking approval for lower doses for patients weighing less than 27 kg is planned for this summer.

Fabrice Chouraqui, Chief Executive Officer of Pharming, commented:

“We are pleased that the FDA has accepted our resubmission, bringing us one step closer to potentially making Joenja available to younger patients living with APDS. These young patients and their families continue to face the significant burden of this rare disease, and there is a clear need for an approved targeted treatment. We look forward to the continued collaboration with the FDA to help bring Joenja to the broader pediatric APDS population as quickly as possible.”

The resubmission is supported by positive data from the open-label, multinational, single-arm Phase III study in children aged 4 to 11 years, which showed improvements over 12 weeks in two clinically relevant hallmarks of APDS, reduced lymphadenopathy and increased naïve B cells, together indicating a correction of the underlying immune defect. The improvements in lymphoproliferation and immunophenotype correction were seen across all dose levels investigated and were consistent with the improvements previously reported in adolescent and adult patients. All treatment emergent adverse events were reported to be mild to moderate in nature. There were no drug related serious adverse events, and all patients completed the 12-week treatment period.

Joenja received approval from the FDA for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023.

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 develop and commercialize a portfolio of 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 & 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

• Pharming announces U.S. FDA acceptance of sNDA resubmission for Joenja_EN_4JUN26 (3)

FAQ

What did Pharming (PHARM) announce about the Joenja sNDA for children with APDS?

Pharming announced FDA acceptance of its resubmitted sNDA for Joenja to treat children aged 4–11 years with APDS. According to Pharming, the filing covers 40 mg and 50 mg twice-daily dosing for pediatric patients weighing at least 27 kg.

When is the FDA PDUFA date for Pharming’s Joenja sNDA in pediatric APDS (PHARM)?

The FDA has set a PDUFA target action date of October 24, 2026 for the Joenja pediatric sNDA. According to Pharming, this date reflects the agency’s timeline to complete its review for children aged 4–11 years with APDS.

What clinical data support Pharming’s Joenja sNDA for children aged 4–11 with APDS?

The resubmission is supported by an open-label Phase III study showing improved lymphadenopathy and increased naïve B cells over 12 weeks. According to Pharming, these changes suggest correction of the underlying immune defect and were consistent across all dose levels investigated.

How was the safety profile of Joenja in Pharming’s pediatric Phase III APDS study?

Joenja’s safety profile in children 4–11 years showed only mild to moderate treatment-emergent adverse events. According to Pharming, no drug-related serious adverse events occurred and all patients completed the 12-week treatment period in the Phase III study.

Could Joenja become the first approved treatment for children with APDS in the U.S.?

If the pediatric sNDA is approved, Joenja would be the first approved U.S. treatment for children with APDS. According to Pharming, Joenja is already FDA-approved for adults and patients 12 years and older, and the filing extends use to ages 4–11.

What are Pharming’s next regulatory plans for Joenja in lighter pediatric APDS patients?

Pharming plans a separate sNDA for lower-dose Joenja in pediatric patients weighing under 27 kg in the second half of 2026. According to Pharming, this aims to broaden Joenja’s potential use across the entire pediatric APDS population.

How does Pharming’s Joenja resubmission address the FDA’s previous Complete Response Letter?

The resubmission includes additional analytical data requested after the January 30, 2026 Complete Response Letter. According to Pharming, these data relate to analytical methods for production batch testing and were discussed in a subsequent Type A meeting with the FDA.