EMA Marketing Authorisation Application for leniolisib changed
to standard review timetable
LEIDEN, Netherlands, Feb. 16,
2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming"
or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR)
announces that the European Medicines Agency's (EMA) Committee for
Human Medicinal Products (CHMP) has decided to shift its assessment
of the Marketing Authorisation Application (MAA) for leniolisib to
a standard review timetable. Leniolisib is an oral selective
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.
Pharming has 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 for
our responses, Pharming expects that CHMP will issue its opinion on
the leniolisib MAA in 2H 2023.
Sijmen de Vries, CEO of Pharming commented:
"We are continuing to work with EMA through the MAA review
process and remain dedicated to seeking regulatory approval of
leniolisib within the European Economic Area. In the US, the FDA's
priority review of the leniolisib NDA remains on track, with a
PDUFA goal date of March 29, 2023. We
remain focused on achieving regulatory approvals and bringing
leniolisib to patients living with APDS worldwide."
Following the grant of accelerated assessment in August 2022, EMA validated Pharming's MAA for
leniolisib for scientific evaluation under an accelerated
assessment by the CHMP in October
2022. The MAA is supported by positive data from a Phase
II/III study of leniolisib, announced on February 2, 2022, which met its co-primary
endpoints of reduction in lymph node size and increase in
percentage of naïve B cells in patients with APDS. Furthermore,
safety data from the study showed that leniolisib was well
tolerated by participants. Also submitted 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 maturation
of white blood cells. 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
hyperactive, immune cells fail to mature and function properly,
leading to immunodeficiency and dysregulation.2,4 APDS
is characterized by severe, 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 suffer a median 7-year
diagnostic delay.7 As APDS is a progressive disease,
this delay may lead to an accumulation of damage over time,
including permanent lung damage and lymphoma.5-8 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. PI3Kδ is expressed
predominately in hematopoietic cells and is essential to normal
immune system function through conversion of
phosphatidylinositol-4-5-trisphosphate (PIP2) to
phosphatidylinositol-3-4-5-trisphosphate (PIP3). Leniolisib
inhibits the production of PIP3 and 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 immune diseases
such as APDS. 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 in patients with APDS.
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 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 their use of terms and
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''could'', ''estimate'', ''expect'', ''goals'', ''intend'',
''may'', "milestones", ''objectives'', ''outlook'', ''plan'',
''probably'', ''project'', ''risks'', "schedule", ''seek'',
''should'', ''target'', ''will'' and similar terms and phrases.
Examples of forward-looking statements may include statements with
respect to 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, 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. 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. 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. Pharming does not undertake any obligation to publicly
update or revise any.
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
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.
For further public information, contact:
Pharming Group, Leiden, The
Netherlands
Michael Levitan, VP Investor Relations & Corporate
Communications
T: +1 (908) 705 1696
Heather Robertson, Investor
Relations & Corporate Communications Manager
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 E: Ethan.Metelenis@precisionvh.com T: +1
(917) 882 9038
EU PR
Dan Caley E: Dan.caley@aprilsix.com T:
+44 (0) 787 546 8942
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