Country for PR: United Kingdom
Contributor: PR Newswire Europe
Wednesday, September 28 2022 - 16:00
Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib
LEIDEN, The Netherlands, Sep 28, 2022, /PRNewswire-AsiaNet/--

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

Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) 
(Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has 
accepted for priority review its New Drug Application (NDA) for leniolisib, an 
oral, selective phosphoinositide 3-kinase delta (PI3Kdelta) inhibitor, to treat 
the rare primary immunodeficiency activated phosphoinositide 3-kinase delta 
syndrome (APDS) in adults and adolescents 12 years of age and older in the US. 
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of 
March 29, 2023, aligned with a Priority Review classification.

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. Those results demonstrated the efficacy of leniolisib over 
placebo with a statistically significant reduction from the baseline size of 
participants’ index lymphadenopathy lesions (p=0.006) and normalization of 
their immune function, as evidenced by an increased proportion of naïve B cells 
from the baseline (p=0.002). Those findings indicate a reduction in disease 
markers associated with APDS, whose clinical hallmarks include significant 
lymphoproliferation and immune dysfunction, as well as increased risk of 
lymphoma. Furthermore, safety data from the study showed that leniolisib was 
well tolerated by participants. Also submitted as part of the application were 
data from a long-term, open-label extension clinical trial including 38 
patients with APDS who were treated with leniolisib for a median of 102 weeks.

Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
"The FDA's acceptance for priority review of Pharming's New Drug Application 
for leniolisib is a milestone that demonstrates our commitment to addressing 
unmet needs for patients with rare diseases. With FDA’s review, leniolisib 
moves further along the regulatory pathway as a potential disease-modifying 
targeted treatment for APDS in adults and adolescents 12 years of age and older 
in the US, 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 with regulatory authorities across the globe, 
to make leniolisib available to immunologists, hematologists, and their APDS 

About Activated Phosphoinositide 3-Kinase delta Syndrome (APDS)
APDS is a rare primary immunodeficiency that affects approximately 1 to 2 
people per million. It is caused by variants in either of two genes, PIK3CD or 
PIK3R1, that regulate maturation of white blood cells. Variants of these genes 
lead to hyperactivity of the PI3Kdelta (phosphoinositide 3-kinase delta) 
pathway.1,2 Balanced signaling in the PI3Kdelta pathway is essential for 
physiological immune function. When this pathway is hyperactive, immune cells 
fail to mature and function properly, leading to immunodeficiency and 
dysregulation.1,3 APDS is characterized by severe, 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 7-year diagnostic delay.6 As APDS is a progressive disease, 
this delay may lead to an accumulation of damage 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 immunomodulating 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 cell 
functions such as proliferation, differentiation, cytokine production, cell 
survival, angiogenesis, and metabolism. Unlike PI3Kalpha and PI3Kbeta, which 
are ubiquitously expressed, PI3Kdalta and PI3Kgaba are expressed primarily in 
cells of hematopoietic origin. The central role of PI3Kdelta in regulating 
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) strongly indicates that PI3Kdelta is a valid and 
potentially effective therapeutic target for several immune diseases. To date, 
leniolisib has been well tolerated during both the Phase 1 first-in-human trial 
in healthy subjects and the Phase II/III registration-enabling study.

About 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 protein replacement therapies and 
precision medicines, including small molecules, biologics, and gene therapies 
that are in early to late-stage development. Pharming is headquartered in 
Leiden, Netherlands, and has employees around the globe who serve patients in 
over 30 markets in North America, Europe, the Middle East, Africa, and 
For more information, visit and find us on LinkedIn

Forward-Looking Statements 
This press release contains forward-looking statements, including with respect 
to 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 the challenges posed by the COVID-19 pandemic to 
the conduct of its business, and Pharming’s expectations regarding its 
projected working capital requirements and cash resources, which 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 
ramifications for the cost thereof; and clinical, scientific, regulatory and 
technical developments. In light of these risks and uncertainties, and other 
risks and uncertainties that are described in Pharming’s 2021 Annual Report and 
the Annual Report on Form 20-F for the year ended December 31, 2021 filed with 
the U.S. Securities and Exchange Commission, 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 thereby. Any 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 release.

Inside Information
This press release relates to 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. 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. 
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 further public information, contact:
Pharming Group, Leiden, The Netherlands
Heather Robertson, Investor Relations & Corporate Communications Manager 
T: +31 71 524 7400

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

Ethan Metelenis
T: +1 (917) 882 9038

Dan Caley
T: +44 (0) 787 546 8942

SOURCE: Pharming Group N.V.