Pharming Group provides updates on EMA regulatory review of
leniolisib MAA and plans to file for UK regulatory approval
Leiden, The Netherlands, November 10,
2023: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that it has
received a Day 180 Second List of Outstanding Issues (LoOI) from
the European Medicines Agency’s (EMA) Committee for Human Medicinal
Products (CHMP) regarding its Marketing Authorisation Application
(MAA) for leniolisib for adult and pediatric patients 12 years of
age and older with APDS, a rare primary immunodeficiency. Based on
the timetable included in the CHMP Day 180 Second LoOI, and
considering the CHMP’s decision to reschedule the Ad-Hoc Expert
Group (AEG) meeting to the end of November, Pharming now expects
the CHMP to issue its opinion on the leniolisib MAA in the first
quarter of 2024.
Pharming now intends to file an MAA with the
U.K. Medicines and Healthcare products Regulatory Agency (MHRA)
through the International Recognition Procedure (IRP) which will
replace the European Commission Decision Recognition Procedure
(ECDRP) from January 1, 2024. Under the IRP, Pharming would file an
MAA for leniolisib on the basis of the US FDA approval in the first
quarter of 2024 and the MHRA would have 110 days from the date the
IRP submission is validated to review and issue its decision.
About Activated Phosphoinositide 3-Kinase δ
Syndrome (APDS)
APDS is a rare primary immunodeficiency that was
first characterized in 2013. APDS is caused by variants in either
one of two identified genes known as PIK3CD or PIK3R1, which are
vital to the development and function of immune cells in the body.
Variants of these genes lead to hyperactivity of the PI3Kδ
(phosphoinositide 3-kinase delta) pathway, which causes immune
cells to fail to mature and function properly, leading to
immunodeficiency and dysregulation1,2,3 APDS is characterized by a
variety of symptoms, including 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, it has been
reported that 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 A definitive
diagnosis can be made through genetic testing. APDS affects
approximately 1 to 2 people per million worldwide.
About leniolisib
Leniolisib is an oral small molecule
phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the
US as the first and only targeted treatment of activated
phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult
and pediatric patients 12 years of age and older. Leniolisib
inhibits the production of
phosphatidylinositol-3-4-5-trisphosphate, which serves as an
important cellular messenger and regulates a multitude of cell
functions such as proliferation, differentiation, cytokine
production, cell survival, angiogenesis, and metabolism. Results
from a randomized, placebo-controlled Phase II/III clinical trial
demonstrated clinical efficacy of leniolisib in the coprimary
endpoints; demonstrating statistically significant impact on immune
dysregulation and normalization of immunophenotype within these
patients, and interim open label extension data has supported the
safety and tolerability of long-term leniolisib administration.8
Leniolisib is currently under regulatory review by the European
Medicines Agency, with plans to pursue further regulatory approvals
in the UK, Canada, Australia and Japan. Leniolisib is also being
evaluated in a Phase III clinical trial in children aged 4 to 11
with APDS, with a further trial planned in children aged 1 to 6
years 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 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 2022 Annual Report and the Annual
Report on Form 20-F for the year ended December 31, 2022, 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
- Lucas CL, et al. Nat Immunol. 2014;15(1):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.
- RAO VK, et al Blood. 2023 Mar 2;141(9):971-983
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
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