Pharming Group provides update on ongoing regulatory review of
leniolisib for the treatment of APDS in the European Union
Leiden,
the Netherlands, May 30,
2024: Pharming Group N.V. (“Pharming” or
“the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces
today an update on the ongoing review of its Marketing
Authorisation Application (MAA) for leniolisib for the treatment of
adult and pediatric patients 12 years of age and older with
activated phosphoinositide 3-kinase delta syndrome (APDS) by the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA).
Following the May 27-30 CHMP meeting, Pharming
received an updated List of Outstanding Issues (LoOI) from the
CHMP. The LoOI affirmed the positive clinical benefit and safety of
leniolisib, in agreement with the assessment by the Ad Hoc Expert
Group (AEG), and included one remaining chemistry, manufacturing
and controls (CMC) request.
The CMC request relates to the definition of
regulatory starting materials used in the manufacturing process for
leniolisib. As Pharming is committed to meeting all of the CHMP’s
specific requirements, additional data and quality controls were
provided and Pharming proposed implementation of the CMC request
post-approval. The CHMP requested that this work be completed
pre-approval and has granted Pharming an extension to January 2026
to submit a response. Pharming has already initiated the
manufacturing activities requested by the CHMP, which it plans to
complete prior to this deadline.
Sijmen de Vries, MD, Chief Executive
Officer of Pharming, commented:“While we are
understandably disappointed by the delay to the European license,
we are pleased that the CHMP determined the clinical benefit of
leniolisib to be positive. The efficacy and safety demonstrated in
clinical trials and ongoing real-world experience, with over 300
patient-years of treatment, support that leniolisib fills an unmet
medical need. We will continue to work closely with the EMA and
CHMP to obtain approval for leniolisib in Europe for people living
with APDS. In the meantime, all our clinical development and early
access programs will continue.”
The MAA for leniolisib was based on findings
from a multinational, triple-blind, placebo-controlled, randomized
Phase II/III clinical trial, which met both its co-primary
endpoints. The trial evaluated efficacy and safety in 31 patients
diagnosed with APDS aged 12 years and older. Also submitted as part
of the application were data from a long-term, open-label extension
clinical trial in which 37 patients received leniolisib for a
median of three years.
Leniolisib is currently available commercially
in the United States. The US Food and Drug Administration (FDA)
approved leniolisib in March 2023, based on its assessment that
leniolisib met clinical and manufacturing standards.
Pharming is maintaining its previously provided
2024 total revenue guidance.
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 leniolisibLeniolisib is
an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ)
inhibitor approved in the US and Israel 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.8,9
Leniolisib is currently under regulatory review in the European
Economic Area, the U.K., Canada and Australia, with plans to pursue
further regulatory approvals in Japan and South Korea. Leniolisib
is also being evaluated in two Phase III clinical trials in
children 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 and biologics.
Pharming is headquartered in Leiden, the 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, commercial, competitive and
technical developments. In light of these risks and uncertainties,
and other risks and uncertainties that are described in Pharming's
2023 Annual Report and the Annual Report on Form 20-F for the year
ended December 31, 2023, 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. 2020 Dec;59(3):323-333.
- Condliffe AM, Chandra A. Front
Immunol. 2018;9:338.
- Rao VK, et al Blood. 2023 Mar
2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol
2024;153:265-74.
For further public information,
contact:Pharming Group, Leiden, The NetherlandsMichael
Levitan, VP Investor Relations & Corporate CommunicationsT: +1
(908) 705 1696E: investor@pharming.com
FTI Consulting, London, UKVictoria Foster
Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam,
The NetherlandsLeon MelensT: +31 6 53 81 64 27E:
pharming@lifespring.nl
US PRChristina RenfroeE:
Christina.Renfroe@precisionvh.com T: +1 (636) 352-7883
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