Marketing authorisation in the European
Economic Area anticipated in H1 2023
LEIDEN, Netherlands,
Oct. 28, 2022 /PRNewswire/ --
Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT
Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing
Authorisation Application (MAA) for leniolisib has been validated
for scientific evaluation under an accelerated assessment by the
European Medicines Agency's (EMA) Committee for Medicinal Products
for Human Use (CHMP). The application, submitted earlier in
October 2022, is for the
investigational drug, leniolisib, an oral, selective
phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment
for activated phosphoinositide 3-kinase delta syndrome (APDS), a
rare primary immunodeficiency, in adolescents and adults 12 years
or older.
In August 2022, Pharming announced
the leniolisib MAA was granted accelerated assessment by EMA's
CHMP. The accelerated assessment reduces the review timeframe from
210 days to 150 days. Upon request, EMA will grant an accelerated
assessment of an MAA if they decide the product is of major
interest for public health, and in particular, from the viewpoint
of therapeutic innovation. Marketing authorisation for leniolisib
in the European Economic Area is anticipated in H1 2023.
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 MAA were data from a long-term, open-label extension
clinical trial in patients with APDS treated with leniolisib.
Anurag Relan, MD, MPH, Chief
Medical Officer of Pharming, commented:
"EMA's validation for review of our MAA under an accelerated
assessment pathway highlights Pharming's ongoing commitment to
advance leniolisib as a targeted treatment for adults and
adolescents 12 years of age or older with APDS. We anticipate that
leniolisib will fill an unmet need for patients with APDS, who
currently rely on supportive therapies to treat their primary
symptoms. This review constitutes a key milestone in
Pharming's effort to give healthcare providers and their patients
global access to leniolisib. We look forward to collaborating with
EMA as needed throughout the regulatory process."
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.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. 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.
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
phrases such as "aim", "ambition", ''anticipate'', ''believe'',
''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 forward-looking statement as a result of new
information, future events or other information.
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. 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.
2019;143(5):1676-1687.
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
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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