Marketing authorisation within the European Economic Area anticipated in H1 2023
LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ — Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) declares at present that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific analysis under an accelerated evaluation by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). The software, submitted earlier in October 2022, is for the investigational drug, leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a therapy for activated phosphoinositide 3-kinase delta syndrome (APDS), a uncommon major immunodeficiency, in adolescents and adults 12 years or older.
In August 2022, Pharming introduced the leniolisib MAA was granted accelerated evaluation by EMA’s CHMP. The accelerated evaluation reduces the evaluation timeframe from 210 days to 150 days. Upon request, EMA will grant an accelerated evaluation of an MAA in the event that they determine the product is of main curiosity for public well being, and particularly, from the perspective of therapeutic innovation. Marketing authorisation for leniolisib within the European Economic Area is anticipated in H1 2023.
The MAA is supported by optimistic 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 dimension and improve in proportion of naïve B cells in sufferers with APDS. 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.
Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
“EMA’s validation for review of our MAA under an accelerated assessment pathway highlights Pharming’s ongoing commitment to advance leniolisib as a targeted treatment for adults and adolescents 12 years of age or older with APDS. We anticipate that leniolisib will fill an unmet need for patients with APDS, who currently rely on supportive therapies to treat their primary symptoms. This review constitutes a key milestone in Pharming’s effort to give healthcare providers and their patients global access to leniolisib. We look forward to collaborating with EMA as needed throughout the regulatory process.”
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a uncommon major immunodeficiency that impacts roughly 1 to 2 individuals per million. APDS is brought on by variants in both of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of those 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 perform. 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 could be related to a wide range of situations, together with different major immunodeficiencies, individuals with APDS are regularly misdiagnosed and undergo 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 injury and lymphoma.4-7 The solely option 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. PI3Kδ is expressed predominately in hematopoietic cells and is crucial to regular immune system perform by conversion of phosphatidylinositol-4-5-trisphosphate (PIP2) to phosphatidylinositol-3-4-5-trisphosphate (PIP3). Leniolisib inhibits the manufacturing of PIP3 and PIP3 serves as an essential mobile messenger activating AKT (by way of PDK1) and regulates a large number of cell features similar 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 features 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 immune illnesses similar to APDS. 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 in sufferers with APDS.
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 modern portfolio of protein alternative therapies and precision medicines, together with small molecules, biologics, and gene therapies which can be in early to late-stage growth. Pharming is headquartered in Leiden, Netherlands, and has staff 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.
Forward-Looking Statements
This press launch could comprise forward-looking statements. Forward-looking statements are statements of future expectations which can be based mostly on administration’s present expectations and assumptions and contain identified and unknown dangers and uncertainties that might trigger precise outcomes, efficiency or occasions to vary materially from these expressed or implied in these statements. These forward-looking statements are recognized by their use of phrases and phrases similar to “aim”, “ambition”, ”anticipate”, ”consider”, ”might”, ”estimate”, ”count on”, ”targets”, ”intend”, ”could”, “milestones”, ”aims”, ”outlook”, ”plan”, ”in all probability”, ”mission”, ”dangers”, “schedule”, ”search”, ”ought to”, ”goal”, ”will” and comparable phrases and phrases. Examples of forward-looking statements could embody statements with respect to timing and progress of Pharming’s preclinical research and scientific trials of its product candidates, Pharming’s scientific and business prospects, and Pharming’s expectations concerning its projected working capital necessities and money sources, which statements are topic to quite a few dangers, uncertainties and assumptions, together with, however not restricted to the scope, progress and enlargement of Pharming’s scientific trials and ramifications for the associated fee thereof; and scientific, scientific, regulatory and technical developments. In mild of those dangers and uncertainties, and different dangers and uncertainties which can 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 might differ materially and adversely from these anticipated or implied thereby. All forward-looking statements contained on this press launch are expressly certified of their entirety by the cautionary statements contained or referred to on this part. Readers mustn’t place undue reliance on forward-looking statements. Any forward-looking statements communicate solely as of the date of this press launch and are based mostly on info out there to Pharming as of the date of this launch. Pharming doesn’t undertake any obligation to publicly replace or revise any forward-looking assertion because of new info, future occasions or different info.
Inside Information
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References
1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.
7. 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
E: [email protected]
T: +1 (917) 882 9038
EU PR:
Dan Caley
E: [email protected]
T: +44 (0) 787 546 8942
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