Application relies on randomized, managed and long-term extension information for leniolisib as a remedy for APDS, a uncommon main immunodeficiency
This submission follows the grant of accelerated evaluation permitting an expedited evaluation for leniolisib from a commonplace 210 days to 150 days
LEIDEN, Netherlands, Oct. 11, 2022 /PRNewswire/ — Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) proclaims that it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a remedy for activated phosphoinositide 3-kinase delta syndrome (APDS), a uncommon main immunodeficiency, in adults and adolescents 12 years or older.
On August 1, 2022, Pharming introduced the leniolisib MAA was granted accelerated evaluation by EMA’s Committee for Medicinal Products for Human Use (CHMP). The accelerated evaluation reduces the evaluation timeframe from 210 days to 150 days. Upon request, the EMA will grant an accelerated evaluation of an MAA in the event that they resolve the product is of main curiosity for public well being, and particularly, from the viewpoint of therapeutic innovation. Marketing authorisation for leniolisib in the EEA is anticipated in H1 2023.
Anurag Relan, Chief Medical Officer of Pharming, commented:
“This MAA submission, under an accelerated regulatory pathway, is an important step towards approval of our second product in the EEA and highlights Pharming’s ongoing commitment to advancing leniolisib as a treatment for patients with APDS. There is a significant unmet need for therapies to improve outcomes for these patients, which, if left untreated, can result in permanent lung damage and lymphoma. Leniolisib has the potential to be the first approved treatment for this rare and orphan-designated disease, and we look forward to continuing our work with key stakeholders to bring this new product to patients.”
The MAA is supported by constructive information from a Phase II/III examine of leniolisib, introduced on February 2, 2022, which met its co-primary endpoints of discount in lymph node measurement and correction of immunodeficiency in the goal inhabitants. Furthermore, security information from the examine confirmed that leniolisib was properly tolerated by contributors. Also submitted as a part of the MAA have been information from a long-term, open-label extension scientific trial in sufferers with APDS handled with leniolisib.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a uncommon main 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 those genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kδ pathway is crucial for physiological immune operate. When this pathway is hyperactive, immune cells fail to mature and performance correctly, main to immunodeficiency and dysregulation.1,3 APDS is characterised by extreme, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these signs could be related to a number of circumstances, together with different main immunodeficiencies, individuals with APDS are often misdiagnosed and undergo a median 7-year diagnostic delay.6 As APDS is a progressive illness, this delay could lead to an accumulation of harm over time, together with everlasting lung injury and lymphoma.4-7 The solely method 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 sophistication IA PI3K with immunomodulating and doubtlessly anti-neoplastic actions. Leniolisib inhibits the manufacturing of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an necessary mobile messenger activating AKT (through PDK1) and regulates a multitude of cell capabilities reminiscent of 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 legitimate and doubtlessly efficient therapeutic goal for a number of immune ailments. 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 international biopharmaceutical firm devoted to remodeling the lives of sufferers with uncommon, debilitating, and life-threatening ailments. Pharming is commercializing and creating an modern portfolio of protein substitute therapies and precision medicines, together with small molecules, biologics, and gene therapies which are in early to late-stage growth. Pharming is headquartered in Leiden, Netherlands, and has workers round 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 comprises forward-looking statements, together with with respect to timing and progress of Pharming’s preclinical research and scientific trials of its product candidates, Pharming’s scientific and business prospects, Pharming’s means to overcome 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 assets, which statements are topic to a variety of dangers, uncertainties and assumptions, together with, however not restricted to the scope, progress and growth of Pharming’s scientific trials and ramifications for the value thereof; and scientific, scientific, regulatory and technical developments. In mild of those dangers and uncertainties, and different dangers and uncertainties which are 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 based mostly on info accessible to Pharming as of the date of this launch.
Inside Information
This press launch relates to the disclosure of knowledge that qualifies, or could have certified, as inside info inside 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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