Pharming Announces US FDA Acceptance of Priority Review of New Drug Application for Leniolisib

The FDA has set a PDUFA deadline of March 29, 2023 for NDA submission based on randomized controlled, long-term extension data for leniolisib as a treatment for APDS, a rare primary immunodeficiency

LEIDEN, The Netherlands, September 28, 2022 /CNW/ — Pharming Group NV (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that the United States Food and Drug Administration (FDA) has accepted for priority review its application New Drug Release (NDA) for leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare phosphoinositide 3-kinase delta (APDS)-activated primary immunodeficiency syndrome (APDS) in adults and adolescents 12 years and older in the United States. The FDA has set a deadline for the Prescription Drug User Fee Act (PDUFA) of March 29, 2023aligned with a priority review classification.

Logo of Pharming Group NV (PRNewsfoto/Pharming Group NV)

Submitted by Pharming on July 29, 2022, the NDA was supported by positive data from a Phase II/III study of leniolisib, which met its co-primary endpoints of reduction in index lymph node size and correction of immunodeficiency in the target population. These results demonstrated the efficacy of leniolisib compared to placebo with a statistically significant reduction from baseline in participants’ index lymphadenopathy lesions (p=0.006) and normalization of their immune function, as evidenced by a proportion increased number of naive B lymphocytes compared to the initial value (p=0.002). These results indicate a reduction in disease markers associated with APDS, the clinical features of which include significant lymphoproliferation and immune dysfunction, as well as an increased risk of lymphoma. In addition, safety data from the study showed that leniolisib was well tolerated by participants. Data from a long-term, open-label, extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median duration of 102 weeks were also submitted as part of the application.

Anurag RelanMD, MPH, Chief Medical Officer of Pharming, commented:
“The FDA’s acceptance of Pharming’s New Drug Application Priority Review for leniolisib is an important milestone that demonstrates our commitment to addressing the unmet needs of rare disease patients. With FDA review, leniolisib is progressing through the regulatory pathway as a potential disease-modifying targeted therapy. for APDS in adults and adolescents 12 years of age and older in the United States, 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 regulatory authorities around the world. globe, to make leniolisib available to immunologists, hematologists and their APDS patients. »

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immune deficiency that affects approximately 1 to 2 people per million. 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 frequently 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.

About Leniolisib
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 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.

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Forward-looking statements
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, as such statements 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 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.

Inside information
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.


  1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.

  2. Elkaim E, and Al. J Allergy Clin Immunol. 2016;138(1):210-218.

  3. Nunes-Santos C, Uzel G, Rosenzweig SOUTH DAKOTA. J Allergy Clin Immunol. 2019;143(5):1676-1687.

  4. Cutter TI, and Al. J Allergy Clin Immunol. 2017;139(2):597-606.

  5. maccari ME, and Al. From the front Immunol. 2018;9:543.

  6. James M, and Al. Wink Rev Allergy Immunol. 2019;May 21.

  7. Condcliffe A M, Chandra A.Before Immunol. 2018;9:338.

For more public information, contact:

Pharmaceutical Group, Leiden, The Netherlands
Heather Robertson, Head of Investor Relations and Corporate Communications
Such. : +31 71 524 7400
E: [email protected]

FTI Consulting, London, UK
Victoria Foster Mitchell/Alex Shaw/Amy Byrne
Such. : +44 203 727 1000

LifeSpring Life Science Communication, Amsterdam, Netherlands
Leon Melens
Tel: +31 6 53 81 64 27
E: [email protected]

Ethan Metelenis
T: +1 (917) 882 9038
E: [email protected]

Dan Caley
Such. : +44 (0) 787 546 8942
Email: [email protected]

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