Pharming announces marketing authorization in the U.K. for Joenja® (leniolisib)
Pharming Group N.V. announces that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
Leniolisib is the first treatment approved in the U.K. specifically for APDS, a rare and progressive primary immunodeficiency. It is currently under evaluation by the National Institute for Health and Care Excellence (NICE) regarding reimbursement within the National Health Service (NHS) in England.
The MHRA evaluated the Marketing Authorisation Application (MAA) for leniolisib through the International Recognition Procedure (IRP) pathway based on the U.S. Food and Drug Administration (FDA) approval received in March 2023. This marks Pharming's third country approval for APDS.
Pharming Group N.V. annuncia che l'
Leniolisib è il primo trattamento approvato nel Regno Unito specificamente per l'APDS, una rara e progressiva immunodeficienza primaria. Attualmente è in fase di valutazione da parte del Istituto Nazionale per la Salute e l'Eccellenza Clinica (NICE) per quanto riguarda il rimborso all'interno del Servizio Sanitario Nazionale (NHS) in Inghilterra.
La MHRA ha valutato la Domanda di Autorizzazione alla Commercializzazione (MAA) per leniolisib attraverso il Procedura di Riconoscimento Internazionale (IRP) basata sull'approvazione ricevuta dalla Food and Drug Administration (FDA) degli Stati Uniti a marzo 2023. Questo segna il terzo paese in cui Pharming ottiene approvazione per l'APDS.
Pharming Group N.V. anuncia que la Agencia Reguladora de Medicamentos y Productos Sanitarios del Reino Unido (MHRA) ha otorgado la autorización de comercialización para Joenja® (leniolisib) para el tratamiento de la síndrome de activación de la fosfoinositida 3-quinasa delta (PI3Kδ) (APDS) en pacientes adultos y pediátricos a partir de 12 años.
Leniolisib es el primer tratamiento aprobado en el Reino Unido específicamente para el APDS, una inmunodeficiencia primaria rara y progresiva. Actualmente está siendo evaluado por el Instituto Nacional para la Salud y la Excelencia en la Atención (NICE) en relación con el reembolso dentro del Servicio Nacional de Salud (NHS) en Inglaterra.
La MHRA evaluó la Solicitud de Autorización de Comercialización (MAA) para leniolisib a través de la Procedimiento de Reconocimiento Internacional (IRP) basado en la aprobación de la Administración de Alimentos y Medicamentos (FDA) de los Estados Unidos recibida en marzo de 2023. Esto marca la tercera aprobación de Pharming para el APDS en otro país.
Pharming Group N.V.는 영국 의약품 및 보건제품 규제청 (MHRA)가 Joenja® (leniolisib)의 상용화 승인을 발표했음을 알립니다. 이 약물은 활성화된 포스파이노시타이드 3 키나제 델타 (PI3Kδ) 증후군 (APDS) 치료를 위해 12세 이상의 성인 및 소아 환자에게 사용됩니다.
Leniolisib는 영국에서 APDS에 대해 구체적으로 승인된 첫 번째 치료제입니다. 이는 드물고 진행성의 일차 면역결핍증입니다. 현재 영국 건강 및 치료 우수 기관 (NICE)에서 영국 국가 의료 서비스 (NHS) 내 환급에 대해 평가 중입니다.
MHRA는 leniolisib의 마케팅 승인 신청서 (MAA)를 국제 인식 절차 (IRP) 경로를 통해 평가하였으며, 이는 2023년 3월에 미국 식품의약국 (FDA)에서 받은 승인을 기반으로 합니다. 이는 Pharming의 APDS에 대한 세 번째 국가 승인을 의미합니다.
Pharming Group N.V. annonce que l'
Leniolisib est le premier traitement approuvé au Royaume-Uni spécifiquement pour l'APDS, une immunodéficience primaire rare et progressive. Il est actuellement en cours d'évaluation par le National Institute for Health and Care Excellence (NICE) en ce qui concerne le remboursement par le Service National de Santé (NHS) en Angleterre.
La MHRA a évalué la Demande d'Autorisation de Mise sur le Marché (MAA) pour leniolisib par le biais de la Procédure de Reconnaissance Internationale (IRP), sur la base de l'approbation obtenue par la Food and Drug Administration (FDA) des États-Unis en mars 2023. Cela constitue la troisième approbation de Pharming pour l'APDS dans un autre pays.
Pharming Group N.V. gibt bekannt, dass die Regulierungsbehörde für Arzneimittel und Gesundheitsprodukte des Vereinigten Königreichs (MHRA) die Marktgenehmigung für Joenja® (leniolisib) zur Behandlung des aktivierten Phosphoinositid-3-Kinase-Delta-Syndroms (PI3Kδ) (APDS) bei erwachsenen und pädiatrischen Patienten ab 12 Jahren erteilt hat.
Leniolisib ist die erste im Vereinigten Königreich speziell für APDS genehmigte Behandlung, eine seltene und progressive primäre Immundefizienz. Derzeit wird das Medikament vom National Institute for Health and Care Excellence (NICE) hinsichtlich der Erstattung im National Health Service (NHS) in England evaluiert.
Die MHRA hat die Anfrage zur Marktgenehmigung (MAA) für leniolisib im Rahmen des International Recognition Procedure (IRP) Prüfverfahrens auf Grundlage der Genehmigung durch die U.S. Food and Drug Administration (FDA) vom März 2023 bewertet. Dies markiert die dritte Genehmigung für APDS durch Pharming in einem anderen Land.
- First treatment approved in the U.K. for APDS
- Marketing authorization granted by MHRA for Joenja® (leniolisib)
- Third country approval for APDS treatment
- Potential for NHS reimbursement pending NICE evaluation
- None.
Insights
The MHRA's approval of Joenja® (leniolisib) for APDS treatment in the UK is a significant development for Pharming and patients. This first-in-class therapy addresses an unmet medical need in a rare immunodeficiency disorder. Key points:
- Joenja® is the first approved treatment specifically for APDS in the UK
- Targets patients 12 years and older, addressing both adult and pediatric populations
- Represents Pharming's third country approval for this indication
- Potential market impact depends on NICE evaluation for NHS reimbursement
While this approval expands Pharming's market reach, the commercial impact will depend on pricing and reimbursement decisions. The rare disease focus aligns with high-value orphan drug strategies, potentially commanding premium pricing. However, the small patient population may limit overall revenue potential. Investors should monitor NICE's evaluation and any subsequent sales traction in the UK market.
For media and investors only
Indicated for adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Leiden, the Netherlands, September 26, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
Leniolisib, an oral, selective PI3Kδ inhibitor, is the first treatment approved in the U.K. specifically for APDS, a rare and progressive primary immunodeficiency. Leniolisib is currently under evaluation by the National Institute for Health and Care Excellence (NICE) regarding reimbursement within the National Health Service (NHS) in England.
Sijmen de Vries, Chief Executive Officer of Pharming, commented:
“The MHRA approval of Joenja®, the first treatment option specifically indicated for APDS, is an important milestone for people in the U.K. living with this debilitating disease. To date, management of APDS has relied on the treatment of the diverse symptoms associated with APDS or for some patients, the need to undergo hematopoietic stem cell transplantation. We are therefore delighted that this product is now approved in the U.K. Today also marks Pharming’s third country approval for APDS, bringing us closer to our goal of becoming a leading global rare disease company dedicated to patient communities with unmet medical needs.”
The MHRA evaluated the Marketing Authorisation Application (MAA) for leniolisib through the International Recognition Procedure (IRP) pathway on the basis of the U.S. Food and Drug Administration (FDA) approval received in March 2023.
Important Safety Information
The full Summary of Product Characteristics (SPC/SmPC) for Joenja® (leniolisib) will be 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 leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the U.S., the U.K. and Israel as the first and only targeted treatment indicated for 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.8,9 Leniolisib is currently under regulatory review in the European Economic Area, Canada and Australia, with plans to pursue further regulatory approvals in Japan and South Korea. Leniolisib is also being evaluated in two Phase III clinical trials in children with 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. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision 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 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, 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.
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
Victoria Foster Mitchell/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 Renfroe
E: Christina.Renfroe@precisionaq.com
T: +1 (636) 352-7883
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