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Indicated for adult and pediatric
patients 12 years of age and older with activated phosphoinositide
3-kinase delta (PI3Kδ) syndrome (APDS)
Leiden, the Netherlands, September 26,
2024: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the United
Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency
(MHRA) has granted marketing authorization for Joenja® (leniolisib)
for the treatment of activated phosphoinositide 3-kinase delta
(PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of
age and older. Leniolisib, an oral, selective PI3Kδ inhibitor, is
the first treatment approved in the U.K. specifically for APDS, a
rare and progressive primary immunodeficiency. Leniolisib is
currently under evaluation by the National Institute for Health and
Care Excellence (NICE) regarding reimbursement within the National
Health Service (NHS) in England.Sijmen de Vries, Chief
Executive Officer of Pharming, commented:“The MHRA
approval of Joenja®, the first treatment option specifically
indicated for APDS, is an important milestone for people in the
U.K. living with this debilitating disease. To date, management of
APDS has relied on the treatment of the diverse symptoms associated
with APDS or for some patients, the need to undergo hematopoietic
stem cell transplantation. We are therefore delighted that this
product is now approved in the U.K. Today also marks Pharming’s
third country approval for APDS, bringing us closer to our goal of
becoming a leading global rare disease company dedicated to patient
communities with unmet medical needs.”The MHRA evaluated the
Marketing Authorisation Application (MAA) for leniolisib through
the International Recognition Procedure (IRP) pathway on the basis
of the U.S. Food and Drug Administration (FDA) approval received in
March 2023.Important Safety Information The full
Summary of Product Characteristics (SPC/SmPC) for Joenja®
(leniolisib) will be available on the MHRA website at
https://products.mhra.gov.uk/.
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 U.S., the U.K. and Israel as the first
and only targeted treatment indicated for 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 III clinical trial
demonstrated statistically significant improvement in the coprimary
endpoints, reflecting a favorable impact on the immune
dysregulation and deficiency seen in 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, 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 forward-looking statement as a result of new
information, future events or other information.
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@precisionaq.com T: +1 (636) 352-7883
- Pharming Group announces marketing authorization
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