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
LEIDEN, The Netherlands, Sept. 28,
2022 /PRNewswire/ -- 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
(PI3Kδ) 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 patients."
About Activated Phosphoinositide 3-Kinase
δ 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
PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2
Balanced signaling in the PI3Kδ 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 PI3Kα and PI3Kβ, which are ubiquitously
expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of
hematopoietic origin. The central role of PI3Kẟ 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 PI3Kẟ 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
Asia-Pacific.
For more information, visit www.pharming.com 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.
References
- Lucas CL, et al. Nat Immunol. 2014;15:88-97.
- Elkaim E, et al. J
Allergy Clin Immunol.
2016;138(1):210-218.
- Nunes-Santos C, Uzel G, Rosenzweig
SD. J Allergy Clin Immunol.
2019;143(5):1676-1687.
- Coulter TI, et al. J Allergy Clin
Immunol. 2017;139(2):597-606.
- Maccari ME, et al. Front
Immunol. 2018;9:543.
- Jamee M, et al. Clin Rev
Allergy Immunol. 2019;May
21.
- 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
E: investor@pharming.com
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: pharming@lifespring.nl
US PR:
Ethan
Metelenis
T: +1 (917) 882 9038
E: Ethan.Metelenis@precisionvh.com
EU PR:
Dan Caley
T: +44 (0) 787 546 8942
E: Dan.caley@aprilsix.com
Logo
- https://mma.prnewswire.com/media/1454235/Pharming_Group_NV_Logo.jpg
View original content to download
multimedia:https://www.prnewswire.com/news-releases/pharming-announces-us-fda-acceptance-for-priority-review-of-its-new-drug-application-for-leniolisib-301634867.html
SOURCE Pharming Group N.V.