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The EMA marketing authorization application for leniolisib has been moved to the standard review schedule.
LEIDEN, The Netherlands, Feb. 16, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has decided to change its assessment of the Application Authorization Agreement (MAA) for leniolisib to a standard review schedule. Leniolisib is a selective oral phosphoinositide 3-kinase delta (PI3Kδ) inhibitor under regulatory review as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years of age and older. And older.
Pharming received the list of questions from EMA which included a request to submit updated data from the ongoing long-term extension study collected after the interim analysis included in the original MAA. Based on the timetable of our responses, Pharming expects the CHMP to issue its opinion on the MAA of leniolisib in the second half of 2023.
Sijmen de Vries, CEO of Pharming, commented:
"We continue to work with EMA through the MAA review process and remain dedicated to seeking regulatory approval for leniolisib within the European Economic Area. In the United States, FDA priority review of leniolisib NDA continues, with a target date." of PDUFA on March 29, 2023. We remain focused on achieving regulatory approvals and bringing leniolisib to patients living with APDS worldwide."
Following the grant of the accelerated evaluation in August 2022, the EMA validated Pharming's MAA for leniolisib for scientific evaluation under an accelerated evaluation of the CHMP in October 2022. The MAA is backed by positive data from a phase II/III study of leniolisib, announced Feb. 2, 2022, that met its coprimary evaluation criteria of reduction in lymph node size and increase in the percentage of virgin B cells in patients with APDS. In addition, the study safety data showed that participants tolerated leniolisib well. Also presented as part of the original MAA were data from a long-term open-label extension clinical trial in patients with APDS treated with leniolisib.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate white blood cell maturation. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.2,3 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is overactive, immune cells do not mature and function properly, leading to immunodeficiency and dysregulation.2,4 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.5,6 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and experience a median diagnostic delay of 7 years.7 Because APDS is a progressive disease, this delay can lead to to an accumulation of damage over time, including damage and lymphoma.5-8 The only way to definitively diagnose this condition is through genetic testing.
Acerca de leniolisib
Leniolisib is a small molecule inhibitor of the delta isoform of the class IA PI3K 110 kDa catalytic subunit. PI3Kδ is expressed predominantly in hematopoietic cells and is essential for the normal functioning of the immune system through the conversion of phosphatidylinositol-4-5-triphosphate (PIP2) to phosphatidylinositol-3-4-5-triphosphate (PIP3). Leniolisib inhibits the production of PIP3, and PIP3 serves as an important cellular messenger that activates AKT (via PDK1) and regulates a multitude of cellular functions including proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are mainly expressed in cells of hematopoietic origin. The central role of PI3Kẟ in the regulation of numerous cellular functions of the adaptive immune system (B cells and, to a lesser extent, T cells), as well as the innate immune system (neutrophils, mast cells, and macrophages), clearly indicates that PI3Kẟ is a target Valid and potentially effective therapeutics for immune diseases such as APDS. To date, leniolisib has been well tolerated during the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration clearance study in patients with APDS.
Acerca de 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 precision medicines and protein replacement therapies, including small molecules, biologics and gene therapies that are in early or late stages of development. Pharming is headquartered in Leiden, The Netherlands, with employees around the world caring for patients in more than 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.
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 the use of terms and phrases such as "target", "ambition", "anticipate", "believe", "could", "estimate", "expect", ''goals'', '' aim'', ''can'', ''milestones'', ''objectives'', ''outlook'', ''plan'', ''probably'', ''project'', ''risks'', "program," "seek," "should," "target," "will," and similar terms and phrases. Examples of forward-looking statements may include statements regarding the 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, the statements of which 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 their cost ramifications yes; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties described in Pharming's 2021 Annual Report and Annual Report on Form 20-F for the year ended December 31, 2021, filed with the Commission on In the United States Securities and Exchange, 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. 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. All 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 press release. Pharming undertakes no obligation to publicly update or revise any.
This press release concerns 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.
1. Rao VK, et al. Blood. 2022. https://doi.org/10.1182/blood.2022018546....
2. Lucas CL, et al. Nat Immunol. 2014;15:88-97.
3. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
4. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
5. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
6. Maccari ME, et al. Front Immunol. 2018;9:543.
7. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.
8. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
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