The FDA has assigned a PDUFA aim date of March 29, 2023 for the NDA submission primarily based on randomized-controlled and long-term extension information for leniolisib as a therapy for APDS, a uncommon major immunodeficiency
LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ — Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) publicizes that the US Food and Drug Administration (FDA) has accepted for precedence evaluation its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to deal with the uncommon major immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older within the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) aim date of March 29, 2023, aligned with a Priority Review classification.
Submitted by Pharming on July 29, 2022, the NDA was supported by constructive information from a Phase II/III examine of leniolisib, which met its co-primary endpoints of discount in index lymph node measurement and correction of immunodeficiency within the goal inhabitants. Those outcomes demonstrated the efficacy of leniolisib over placebo with a statistically important discount from the baseline measurement of contributors’ index lymphadenopathy lesions (p=0.006) and normalization of their immune operate, as evidenced by an elevated proportion of naïve B cells from the baseline (p=0.002). Those findings point out a discount in illness markers related to APDS, whose medical hallmarks embrace important lymphoproliferation and immune dysfunction, in addition to elevated threat of lymphoma. Furthermore, security information from the examine confirmed that leniolisib was properly tolerated by contributors. Also submitted as half of the applying have been information from a long-term, open-label extension medical trial together with 38 sufferers with APDS who have been handled with leniolisib for a median of 102 weeks.
Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
“The FDA’s acceptance for priority review of Pharming’s New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases. With FDA’s review, leniolisib moves further along the regulatory pathway as a potential disease-modifying targeted treatment for APDS in adults and adolescents 12 years of age and older in the US, who currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy. We look forward to continuing to work closely with the FDA, as well as with regulatory authorities across the globe, to make leniolisib available to immunologists, hematologists, and their APDS patients.”
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a uncommon major immunodeficiency that impacts roughly 1 to 2 individuals per million. It is attributable to variants in both of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes result in hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling within the PI3Kδ pathway is crucial for physiological immune operate. When this pathway is hyperactive, immune cells fail to mature and performance correctly, resulting in immunodeficiency and dysregulation.1,3 APDS is characterised by extreme, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these signs will be related to a spread of circumstances, together with different major immunodeficiencies, individuals with APDS are incessantly misdiagnosed and endure a median 7-year diagnostic delay.6 As APDS is a progressive illness, this delay could result in an accumulation of harm over time, together with everlasting lung harm and lymphoma.4-7 The solely technique to definitively diagnose this situation is thru genetic testing.
About Leniolisib
Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and probably anti-neoplastic actions. Leniolisib inhibits the manufacturing of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an vital mobile messenger activating AKT (through PDK1) and regulates a mess of cell capabilities equivalent to proliferation, differentiation, cytokine manufacturing, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, that are ubiquitously expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of hematopoietic origin. The central function of PI3Kẟ in regulating quite a few mobile capabilities of the adaptive immune system (B-cells and, to a lesser extent, T cells) in addition to the innate immune system (neutrophils, mast cells, and macrophages) strongly signifies that PI3Kẟ is a sound and probably efficient therapeutic goal for a number of immune illnesses. To date, leniolisib has been properly tolerated throughout each the Phase 1 first-in-human trial in wholesome topics and the Phase II/III registration-enabling examine.
About Pharming Group N.V.
Pharming Group N.V. (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) is a worldwide biopharmaceutical firm devoted to remodeling the lives of sufferers with uncommon, debilitating, and life-threatening illnesses. Pharming is commercializing and growing an progressive portfolio of protein substitute therapies and precision medicines, together with small molecules, biologics, and gene therapies which might be in early to late-stage growth. Pharming is headquartered in Leiden, Netherlands, and has workers across the globe who serve sufferers in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
For extra info, go to www.pharming.com and discover us on LinkedIn
Forward-Looking Statements
This press launch incorporates forward-looking statements, together with with respect to timing and progress of Pharming’s preclinical research and medical trials of its product candidates, Pharming’s medical and business prospects, Pharming’s potential to beat the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming’s expectations concerning its projected working capital necessities and money sources, which statements are topic to a quantity of dangers, uncertainties and assumptions, together with, however not restricted to the scope, progress and growth of Pharming’s medical trials and ramifications for the fee thereof; and medical, scientific, regulatory and technical developments. In mild of these dangers and uncertainties, and different dangers and uncertainties which might be described in Pharming’s 2021 Annual Report and the Annual Report on Form 20-F for the 12 months ended December 31, 2021 filed with the U.S. Securities and Exchange Commission, the occasions and circumstances mentioned in such forward-looking statements could not happen, and Pharming’s precise outcomes may differ materially and adversely from these anticipated or implied thereby. Any forward-looking statements converse solely as of the date of this press launch and are primarily based on info obtainable to Pharming as of the date of this launch.
Inside Information
This press launch pertains to the disclosure of info that qualifies, or could have certified, as inside info throughout the which means of Article 7(1) of the EU Market Abuse Regulation.
References
- Lucas CL, et al. Nat Immunol. 2014;15:88-97.
- 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. 2019;May 21.
- Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
For additional public info, contact:
Pharming Group, Leiden, The Netherlands
Heather Robertson, Investor Relations & Corporate Communications Manager
T: +31 71 524 7400
E: [email protected]
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: [email protected]
US PR:
Ethan Metelenis
T: +1 (917) 882 9038
E: [email protected]
EU PR:
Dan Caley
T: +44 (0) 787 546 8942
E: [email protected]
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