Pharming Group Receives Accelerated Review in Europe for Leniolisib for the Treatment of Rare Immunodeficiency, APDS
EMA accelerated review allows for a shorter review period for leniolisib from the standard 210 days to 150 days
Pharming is on track to file its marketing authorization application for leniolisib in H2 2022
LEIDEN, the Netherlands, August 1, 2022 /PRNewswire/ — Pharming Group NV (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) granted an accelerated assessment for the marketing authorization application (MA) for leniolisib. Leniolisib has been studied for the treatment of activated PI3K delta syndrome (APDS), a rare primary immune deficiency, in adults and adolescents aged 12 years or older in the European Economic Area (EEA). Pharming is on track and expects to submit its marketing authorization for leniolisib to the EMA in October 2022.
Accelerated assessment reduces the time of CHMP to consider a MAA of 210 days to 150 days. The EMA will grant, on request, an accelerated assessment of an MA if it decides that the product is of major interest for public health and therapeutic innovation.
The clinical development of leniolisib includes positive data from a Phase II/III study of the product, which met its two primary endpoints in the target patient population, namely assessed reduction in lymph node size and correction of immunodeficiency. The primary efficacy results demonstrated the clinical efficacy of leniolisib compared to placebo with a statistically significant reduction from baseline in the transformed log10 sum of the product of diameters (SPD) in index lymphadenopathy lesions (p=0 .0012) and a normalization of immune dysfunction, as evidenced by an increase in the proportion of naive B lymphocytes compared to baseline (p
In the study, leniolisib was generally well tolerated, with the majority of adverse events reported in both treatment groups being classified as mild. No adverse events led to discontinuation of study treatment, no deaths occurred, and the incidence of serious adverse events (SAEs) was lower in the leniolisib group than in the placebo group. None of the SAEs were suspected to be related to the study treatment.
Anurag RelanChief Medical Officer of Pharming, commented:
“Acceptance of accelerated regulatory review for leniolisib underscores the high unmet need for patients with APDS, as the product is potentially the first approved treatment for this rare disease. This is an important step for the APDS community and for Pharming and builds on successful Phase II/III data, which we first reported in February 2022. We remain focused on advancing leniolisib through the regulatory review process, with our marketing authorization pending in October of this year, as we seek to make this important new product available to immunologists, hematologists and their patients in Europe.”
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immune deficiency that affects approximately one to two people in a million. Also known as PASLI, it is caused by variants in one of two genes, PIK3CD Where PIK3R1, which regulate the maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1.2 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is overactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1.3 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity and enteropathy.4.5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are often misdiagnosed and suffer a median diagnostic delay of 7 years.6 Because APDS is a progressive disease, this delay can cause damage to accumulate over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.
Leniolisib is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulatory and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT (via PDK1) and regulates a multitude of cellular functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis and metabolism. Unlike PI3Kα and PI3Kβ, which are expressed ubiquitously, PI3Kẟ and PI3Kγ are expressed mainly in cells of hematopoietic origin. The central role of PI3Kẟ in the regulation of many 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) strongly indicates that PI3Kẟ is a therapeutic target. valid and potentially effective for several immune diseases. To date, leniolisib has been well tolerated in the first-in-man phase 1 trial in healthy subjects and the registration-based phase II/III study.
About Pharming Group NV
Pharming Group NV (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 commercializes and develops an innovative portfolio of protein replacement therapies and precision drugs, including small molecules, biologics and gene therapies in early to late-stage development. Pharming is headquartered in Leiden, Netherlandsand has employees worldwide serving patients in more than 30 markets in North America, Europethe Middle East, Africaand Asia Pacific. For more information, visit www.pharming.com.
This press release contains forward-looking statements, including with respect to the timing and progress of Pharming’s preclinical studies and clinical trials of its product candidates, Pharming’s clinical and commercial prospects, Pharming’s ability to overcome posed by the COVID-19 pandemic to the conduct of its business, and Pharming’s expectations regarding its working capital requirements and cash resources, such statements being 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 ramifications for the cost thereof; 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 United States Securities and Exchange Commission, the events and circumstances discussed in these forward-looking statements may not occur, and Pharming’s actual results may differ materially and adversely from those anticipated or implied. 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.
This press release concerns the disclosure of information which is or could have been classified as inside information within the meaning of Article 7(1) of the European Market Abuse Regulation.
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3. Nunes-Santos C, et al. 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. Immunol before. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2020;59(3):323-333.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
SOURCE Pharming Group SA