Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application for leniolisib in children with APDS aged 4 to 11 years
Pharming Group (NASDAQ:PHAR) announced that the FDA has accepted its supplemental New Drug Application (sNDA) for leniolisib (Joenja®) with Priority Review status. The application seeks approval for treating children aged 4-11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
The sNDA is supported by positive data from a Phase III study showing improvements in lymphadenopathy and increased naïve B cells over 12 weeks. The FDA has set a PDUFA target date of January 31, 2026. If approved, leniolisib would become the first treatment available for APDS patients under 12 years old globally. The drug is already approved for patients 12 years and older since March 2023.
Pharming Group (NASDAQ:PHAR) ha annunciato che la FDA ha accettato la sua domanda supplementare di nuovo medicinale (sNDA) per leniolisib (Joenja®) con stato di revisione prioritaria. La domanda mira all'approvazione per il trattamento dei bambini di 4-11 anni con la sindrome attivata dalla fosfoinositide-3-kinasi delta (APDS), una rara immunodeficienza primaria.
La sNDA è supportata da dati positivi di uno studio di fase III che mostrano miglioramenti nell'adénopatia linfonodale e un aumento delle cellule B naive per 12 settimane. La FDA ha fissato una data obiettivo PDUFA del 31 gennaio 2026. Se approvato, il leniolisib diventerebbe il primo trattamento disponibile per i pazienti APDS con meno di 12 anni a livello globale. Il farmaco è già stato approvato per i pazienti di 12 anni e oltre dal marzo 2023.
Pharming Group (NASDAQ:PHAR) anunció que la FDA ha aceptado su solicitud suplementaria de nuevo fármaco (sNDA) para leniolisib (Joenja®) con estatus de Revisión Prioritaria. La solicitud busca la aprobación para tratar a niños de 4 a 11 años con el síndrome activado de fosfoinositide-3-quinasa delta (APDS), una rara inmunodeficiencia primaria.
La sNDA está respaldada por datos positivos de un estudio de fase III que muestran mejoras en la linfadenopatía y un aumento de células B naive a lo largo de 12 semanas. La FDA ha establecido una fecha objetivo PDUFA del 31 de enero de 2026. Si se aprueba, el leniolisib sería el primer tratamiento disponible para pacientes con APDS menores de 12 años a nivel mundial. El fármaco ya está aprobado para pacientes de 12 años en adelante desde marzo de 2023.
Pharming Group (NASDAQ:PHAR)가 FDA가 leniolisib (Joenja®)에 대한 보충 신약 신청(sNDA)을 우선 심사(Priority Review) 상태로 수락했다고 발표했습니다. 이 신청은 4-11세의 활성화된 포스포이소시티드-3-키나아제 델타(APDS) 증후군을 가진 소아의 치료를 위한 승인을 목표로 하며, 이는 희귀한 일차 면역결핍 질환입니다.
이번 sNDA는 12주 동안 림프선 비대의 개선과 초기화된 B세포 증가를 보여주는 3상(Phase III) 연구의 긍정적 데이터를 지지합니다. FDA는 PDUFA 목표일 2026년 1월 31일을 설정했습니다. 승인되면 leniolisib는 전 세계적으로 12세 미만 APDS 환자를 위한 최초의 치료제가 될 것입니다. 이 약은 이미 2023년 3월부터 12세 이상 환자들에게 승인되어 있습니다.
Pharming Group (NASDAQ:PHAR) a annoncé que la FDA a accepté sa demande supplétive de nouveau médicament (sNDA) pour leniolisib (Joenja®) avec le statut de révision prioritaire. La demande vise l'approbation du traitement des enfants de 4 à 11 ans atteints du syndrome activé de la phosphoinositide 3-kinase delta (APDS), une déficience immunitaire primaire rare.
La sNDA est soutenue par des données positives d'une étude Phase III montrant des améliorations de la lymphadénopathie et une augmentation des lymphocytes B naïfs sur 12 semaines. La FDA a fixé une date cible PDUFA du 31 janvier 2026. Si elle est approuvée, le leniolisib deviendrait le premier traitement disponible pour les patients APDS de moins de 12 ans dans le monde. Le médicament est déjà approuvé pour les patients âgés de 12 ans et plus depuis mars 2023.
Pharming Group (NASDAQ:PHAR) kündigte an, dass die FDA seinen Ergänzungsantrag auf Zulassung eines neuen Medikaments (sNDA) für leniolisib (Joenja®) mit dem Status Priority Review akzeptiert hat. Der Antrag zielt auf die Genehmigung zur Behandlung von Kindern im Alter von 4–11 Jahren mit dem aktivierten Phosphoinositid-3-Kinase-Delta-Syndrom (APDS), einer seltenen primären Immundefizienz.
Die sNDA wird von positiven Daten einer Phase-III-Studie gestützt, die über 12 Wochen Verbesserungen der Lymphadenopathie und eine Zunahme naïver B-Zellen zeigen. Die FDA hat ein PDUFA-Zieltermin am 31. Januar 2026 festgelegt. Falls genehmigt, wäre Lenolisib weltweit die erste Behandlung für APDS-Patienten unter 12 Jahren. Das Medikament ist bereits seit März 2023 für Patienten ab 12 Jahren zugelassen.
Pharming Group (بورصة ناسداك: PHAR) أعلنت أن إدارة الغذاء والدواء الأمريكية (FDA) قبلت طلبها المكمل لدواء جديد (sNDA) لـ leniolisib (Joenja®) بوضع المراجعة الأولوية. يهدف الطلب إلى الموافقة على العلاج للأطفال الذين تتراوح أعمارهم بين 4 و11 عامًا المصابين بمتلازمة فاعلة من إنزيم فوسفوإيزويدا-3 كيناز دلتا (APDS)، وهو نقص مناعي أولي نادر.
يستند sNDA إلى بيانات إيجابية من دراسة المرحلة الثالثة التي أظهرت تحسنًا في Contract lymphadenopathy وزيادة في الخلايا B الساذجة على مدى 12 أسبوعًا. حدّدت FDA تاريخ هدف PDUFA وهو 31 يناير 2026. إذا تمت الموافقة، سيكون leniolisib أول علاج متاح لمرضى APDS دون سن 12 عامًا على مستوى العالم. الدواء مُعتمد بالفعل للمرضى الذين تبلغ أعمارهم 12 عامًا فما فوق منذ مارس 2023.
Pharming Group (NASDAQ:PHAR)宣布，FDA 已接受其用于 leniolisib（Joenja®） 的补充新药申请（sNDA），并给予优先审评状态。该申请寻求批准用于治疗4至11岁、患有活化型磷脂酰肌醇-3-激酶δ综合征(APDS)的儿童，这是一种罕见的原发性免疫缺陷疾病。
该 sNDA 得到来自III期研究的积极数据支持，显示在12周内淋巴结肿大改善且初始B细胞增加。FDA 已设定PDUFA 目标日期为2026年1月31日。如获批准，leniolisib 将成为全球范围内12岁以下 APDS 患者的首个治疗药物。该药物自2023年3月起已获准用于12岁及以上的患者。
- FDA granted Priority Review status, potentially expediting the approval process
- Phase III trial showed positive results in improving key disease markers
- If approved, leniolisib would be the first treatment for APDS in children aged 4-11
- Drug already approved for patients 12 and older, demonstrating established safety profile
- None.
Insights
FDA's Priority Review for Pharming's leniolisib in children could significantly expand treatment options for rare APDS immunodeficiency by January 2026.
The FDA's acceptance of Pharming Group's supplemental New Drug Application (sNDA) with Priority Review status represents a significant regulatory advancement for leniolisib (Joenja®) in treating children aged 4-11 with activated phosphoinositide 3-kinase delta syndrome (APDS). This rare primary immunodeficiency currently has no approved treatments for children under 12 globally, making this potential approval particularly meaningful.
The Priority Review designation is especially noteworthy as it's granted only when the FDA recognizes a therapy could offer substantial improvements in the treatment of serious conditions. This accelerated pathway reduces the standard review time from 10 months to 6 months, with a PDUFA target action date of January 31, 2026.
The application is supported by compelling Phase III data demonstrating improvements in two key disease markers: reduced lymphadenopathy (swollen lymph nodes) and increased naïve B cells, indicating correction of the underlying immune dysfunction. The study's single-arm design is appropriate given the rarity of APDS and the absence of standard treatments.
For Pharming, this regulatory progress builds on their March 2023 approval of leniolisib for patients 12 and older, potentially expanding their market to younger patients where early intervention could significantly alter disease progression. If approved, leniolisib would become the first-in-class therapy for this younger population with APDS, addressing critical unmet needs in children who typically begin experiencing symptoms in early childhood.
- If approved, leniolisib will be first and only treatment indicated for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency
- Decision based on positive data from multinational Phase III study in children aged 4 to 11 years with APDS
- PDUFA target action date of January 31, 2026
Leiden, the Netherlands, October 1, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext: PHARM; Nasdaq: PHAR) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking approval for 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. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026.
The sNDA submitted to the FDA is based on positive data from the 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 the condition, reduced lymphadenopathy and increased naïve B cells, together indicating a correction of the underlying immune defect. The submission also included safety data from 8 months of treatment.
The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements in effectiveness or safety of the treatment, prevention, or diagnosis of serious conditions.1 There are no approved treatments for children with APDS under the age of 12 years globally.
Fabrice Chouraqui, Chief Executive Officer of Pharming, commented:
“APDS is a rare, complex, and progressive primary immunodeficiency. Typically, it begins in early childhood causing immune dysregulation, recurrent infections and potentially permanent lung damage and lymphoma. Early access to targeted therapies has the potential to change the trajectory of the disease for young patients. Today’s Priority Review designation marks a significant step for children aged 4-11 in the U.S. living with APDS.”
Leniolisib, marketed under the brand name Joenja® in the U.S., received approval from the U.S. 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 dysregulation2,3,4 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.5,6 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.7 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.5-8 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
About leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the U.S., U.K., Australia and Israel as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 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 interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.9,10 Leniolisib is currently under regulatory review in the European Economic Area, Japan, Canada and several other countries for APDS. Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS and 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
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References
- FDA. Priority Review. Available at: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review Accessed October 2025.
- Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.
- Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
- Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
- Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
- Maccari ME, et al. Front Immunol. 2018;9:543.
- Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.
- Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
- Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
For further public information, contact:
Pharming Group, Leiden, the Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
Saskia Mehring, Corporate Communications Manager
T: +31 6 28 32 60 41
LifeSpring Life Sciences Communication, Amsterdam, the Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
US PR
Ethan Metelenis
T: +1 (917) 882-9038
E: Ethan.Metelenis@precisionaq.com
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FAQ
What is the PDUFA date for Pharming's leniolisib sNDA for children with APDS?
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Is leniolisib already approved for any age group with APDS?
What is the significance of Priority Review status for Pharming's leniolisib sNDA?
Are there any currently approved treatments for APDS in children under 12?
