Pharming announces marketing authorization in the U.K. for Joenja®
(leniolisib)
For media and investors
only
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 leniolisib
Leniolisib 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 Netherlands
Michael Levitan, VP Investor Relations & Corporate
Communications
T: +1 (908) 705 1696
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
Christina Renfroe
E: Christina.Renfroe@precisionaq.com
T: +1 (636) 352-7883
- Pharming Group announces marketing authorization
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