Pharming receives positive recommendation from NICE for Joenja®▼(leniolisib) as a treatment for APDS
Pharming Group (EURONEXT: PHARM/Nasdaq: PHAR) has received a positive recommendation from the National Institute for Health and Care Excellence (NICE) for Joenja® (leniolisib) in England and Wales. The drug is now the first and only medicine specifically approved for treating APDS (activated phosphoinositide 3-kinase delta syndrome) within the NHS.
The recommendation, based on Phase III clinical trial data, covers both adult and pediatric patients 12 years and older. The trials demonstrated significant improvements in immune deficiency and dysregulation compared to placebo, with maintained benefits during long-term treatment. The drug will be immediately available in England through the Innovative Medicines Fund, while Welsh availability is expected within three months through NHS specialist centers.
Pharming Group (EURONEXT: PHARM/Nasdaq: PHAR) ha ricevuto una raccomandazione positiva dal National Institute for Health and Care Excellence (NICE) per Joenja® (leniolisib) in Inghilterra e Galles. Il farmaco è ora il primo e unico medicinale specificamente approvato per il trattamento della APDS (sindrome da attivazione della fosfoinositide 3-chinasi delta) all'interno del NHS.
La raccomandazione, basata sui dati della fase III degli studi clinici, riguarda sia pazienti adulti che pediatrici di età pari o superiore a 12 anni. Gli studi hanno dimostrato miglioramenti significativi nella carenza immunitaria e nella disregolazione rispetto al placebo, con benefici mantenuti durante il trattamento a lungo termine. Il farmaco sarà immediatamente disponibile in Inghilterra tramite l'Innovative Medicines Fund, mentre la disponibilità in Galles è prevista entro tre mesi attraverso i centri specialistici del NHS.
Pharming Group (EURONEXT: PHARM/Nasdaq: PHAR) ha recibido una recomendación positiva del National Institute for Health and Care Excellence (NICE) para Joenja® (leniolisib) en Inglaterra y Gales. El medicamento es ahora el primero y único específicamente aprobado para tratar el APDS (síndrome de activación de la fosfoinositol 3-quinasa delta) dentro del NHS.
La recomendación, basada en datos del ensayo clínico de fase III, cubre tanto a pacientes adultos como pediátricos de 12 años o más. Los ensayos demostraron mejoras significativas en la deficiencia y desregulación inmunitaria en comparación con el placebo, con beneficios mantenidos durante el tratamiento a largo plazo. El medicamento estará disponible de inmediato en Inglaterra a través del Innovative Medicines Fund, mientras que se espera su disponibilidad en Gales en un plazo de tres meses a través de centros especializados del NHS.
Pharming Group (EURONEXT: PHARM/Nasdaq: PHAR)는 영국과 웨일스에서 Joenja® (leniolisib)에 대해 국가보건임상우수연구원(NICE)으로부터 긍정적인 권고를 받았습니다. 이 약물은 NHS 내에서 APDS(활성화된 포스포이노시티드 3-키나제 델타 증후군)를 치료하기 위해 특별히 승인된 최초이자 유일한 약입니다.
이 권고는 3상 임상시험 데이터를 기반으로 하며, 12세 이상 성인 및 소아 환자를 대상으로 합니다. 임상시험에서는 위약 대비 면역 결핍 및 조절 장애에서 유의미한 개선이 나타났으며, 장기 치료 중에도 효과가 유지되었습니다. 이 약물은 영국에서 혁신 의약품 기금을 통해 즉시 이용 가능하며, 웨일스에서는 NHS 전문 센터를 통해 3개월 이내에 제공될 예정입니다.
Pharming Group (EURONEXT : PHARM/Nasdaq : PHAR) a reçu une recommandation positive du National Institute for Health and Care Excellence (NICE) pour Joenja® (leniolisib) en Angleterre et au Pays de Galles. Ce médicament est désormais le premier et le seul approuvé spécifiquement pour traiter le syndrome APDS (syndrome d'activation de la phosphoinositide 3-kinase delta) au sein du NHS.
La recommandation, basée sur les données des essais cliniques de phase III, concerne les patients adultes et pédiatriques âgés de 12 ans et plus. Les essais ont démontré des améliorations significatives de la déficience immunitaire et de la dysrégulation par rapport au placebo, avec des bénéfices maintenus lors d'un traitement à long terme. Le médicament sera immédiatement disponible en Angleterre via le Innovative Medicines Fund, tandis que sa disponibilité au Pays de Galles est prévue dans les trois mois via les centres spécialisés du NHS.
Pharming Group (EURONEXT: PHARM/Nasdaq: PHAR) hat vom National Institute for Health and Care Excellence (NICE) eine positive Empfehlung für Joenja® (leniolisib) in England und Wales erhalten. Das Medikament ist nun das erste und einzige speziell für die Behandlung von APDS (aktiviertes Phosphoinositid-3-Kinase-Delta-Syndrom) im NHS zugelassene Arzneimittel.
Die Empfehlung basiert auf Daten aus der Phase-III-Studie und umfasst sowohl erwachsene als auch pädiatrische Patienten ab 12 Jahren. Die Studien zeigten signifikante Verbesserungen bei Immundefizienz und Dysregulation im Vergleich zu Placebo, mit anhaltenden Vorteilen während der Langzeitbehandlung. Das Medikament wird in England sofort über den Innovative Medicines Fund verfügbar sein, während die Verfügbarkeit in Wales innerhalb von drei Monaten über NHS-Fachzentren erwartet wird.
- First and only approved treatment for APDS in NHS system
- Phase III trials showed significant clinical improvements vs placebo
- Immediate patient access in England through Innovative Medicines Fund
- Positive clinical trial results maintained during long-term treatment
- None.
Insights
NICE's positive recommendation for Joenja enables immediate NHS reimbursement, establishing Pharming's first-in-class treatment in the valuable UK rare disease market.
The positive NICE recommendation for Joenja® (leniolisib) represents a crucial market access milestone for Pharming. This decision ensures NHS reimbursement in England and Wales for patients 12 years and older with activated phosphoinositide 3-kinase delta syndrome (APDS), an ultra-rare primary immunodeficiency.
Securing reimbursement under NICE's Highly Specialised Technologies pathway - specifically designed for very rare conditions - is particularly significant. For rare disease treatments like Joenja, obtaining reimbursement approval is often as challenging as regulatory clearance itself. The immediate availability through England's Innovative Medicines Fund eliminates the typical access delays that can hamper commercial uptake.
The clinical evidence supporting this recommendation is compelling. Phase III data demonstrated significant improvements in both immune deficiency and immune dysregulation compared to placebo, with benefits maintained during longer-term treatment in the extension trial. These clinical outcomes directly address the fundamental cause of APDS rather than merely managing symptoms.
As the first and only medicine specifically developed for APDS, Pharming has established a first-mover advantage in this therapeutic space. The endorsements from both clinical experts and patient advocacy organizations further validate the unmet need this treatment addresses. Professor Savic's statement highlighting the drug's "real benefits" for patients with this "complex and progressive condition" underscores its clinical value.
This UK reimbursement decision may establish a favorable precedent for Pharming's ongoing market access negotiations in other European territories, potentially accelerating the commercial pathway for Joenja across multiple markets.
For media and investors only
Joenja® is the first and only medicine specifically for APDS to be reimbursed within the NHS
Recommended for adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Leiden, the Netherlands, April 23, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the National Institute for Health and Care Excellence (NICE) has issued positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the National Health Service (NHS) in England and Wales for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
The NICE recommendation is based on the totality of the data, including the Phase III clinical trial evaluating leniolisib, an oral selective PI3Kδ inhibitor, in patients with APDS, a rare and progressive primary immunodeficiency. In the primary analysis, treatment with leniolisib led to significant improvements in immune deficiency and immune dysregulation compared to placebo. The Phase III trial demonstrated an improvement in clinical outcomes, which was maintained during longer-term treatment within the open-label extension trial.
Fabrice Chouraqui, Chief Executive Officer of Pharming, said:
“This positive recommendation for Joenja® from NICE is a significant milestone for patients living with APDS in England and Wales. It underscores Pharming’s commitment to work closely with regulatory and reimbursement authorities, the medical immunology community, and patient associations to bring this first targeted treatment to patients who may benefit.”
Professor Sinisa Savic, Professor of Clinical Immunology at St James’s University Hospital, said:
“Data from clinical trials demonstrate that leniolisib provides real benefits for patients with APDS, a rare primary immunodeficiency. APDS is a complex and progressive condition that leads to immune dysregulation and recurrent infections, significantly impacting patients' quality of life. The recommendation by NICE, which allows this targeted therapy to be prescribed on the NHS, marks an important step forward in improving treatment options for individuals with this condition.”
Dr Susan Walsh, Chief Executive Officer at Immunodeficiency UK, said:
“This is a hugely welcomed decision that will make a massive difference to the lives of people with APDS, and their families, by potentially allowing them to have a better quality of life. With leniolisib, we now have a targeted treatment available that addresses the fundamental cause of the immune system problems experienced in APDS. This is a huge leap forward. We are grateful to Pharming for working with NICE to make this drug available via the NHS. Together, they have shown a real commitment to providing people affected by ultra-rare, immune system conditions the treatments they need.”
The final NICE recommendation aligns with the U.K. Medicines and Healthcare products Regulatory Authority (MHRA) approval and falls under NICE’s Highly Specialised Technologies (HST) pathway, which evaluates treatments for very rare and severe diseases. Leniolisib is now available for use and funded in England through the Innovative Medicines Fund, ensuring immediate patient access. In Wales, leniolisib is expected to be funded within the next three months through the NHS in specialist centers.
Important Safety Information
The full Summary of Product Characteristics (SPC/SmPC) for Joenja® (leniolisib) is available on the MHRA website at https://products.mhra.gov.uk/.
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 dysregulation1,2,3 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.
About Joenja® (leniolisib)
Joenja® (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. 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 interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.8,9 Leniolisib is currently under regulatory review in the European Economic Area, Canada and several other countries for APDS, with plans to pursue regulatory approval in Japan. 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 commercializing and developing a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
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
- 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
E: investor@pharming.com
FTI Consulting, London, UK
Simon Conway/Alex Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, the Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
US PR
Christina Skrivan
T: +1 (917) 882 9038
E: Christina.Skrivan@precisionaq.com
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FAQ
What is the significance of NICE's recommendation for Joenja (PHARM) in treating APDS?
How effective was Joenja (PHARM) in Phase III clinical trials for APDS treatment?
When will Joenja (PHARM) be available to NHS patients in England and Wales?
What age groups are eligible for Joenja (PHARM) treatment under the NHS recommendation?
