APDS (activated phosphoinositide 3-kinase delta (PI3Kδ)
syndrome) is a rare and progressive primary immunodeficiency
Joenja® is a targeted treatment of APDS for adult and pediatric
patients 12 years of age and older
Joenja® is expected to launch in the US in early April
Pharming will host a conference call for investors and analysts
on March 27 at 14:00 CEST/08:00
EST
LEIDEN, The Netherlands, March 24,
2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming"
or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR)
announces that the US Food and Drug Administration (FDA) has
approved 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. Joenja®, an oral,
selective PI3Kδ inhibitor, is the first and only treatment approved
in the US for APDS, a rare and progressive primary
immunodeficiency. The FDA evaluated the Joenja® application for
APDS under Priority Review, which is granted to therapies that have
the potential to provide significant improvements in the treatment,
diagnosis or prevention of serious conditions. Joenja® is expected
to launch in the US in early April and will be available for
shipment in mid-April.
Dr. Eveline Wu, MD, MSCR,
Division Chief, Paediatric Rheumatology & Associate Professor
of Paediatric Rheumatology and Allergy/Immunology at The
University of North Carolina School of
Medicine, said:
"The FDA approval of Joenja® is an exciting moment for the
APDS community and offers to transform the treatment pathway for
patients and families affected by this rare disease. This approval
means that they will, for the first time, have access to an
approved treatment, which has the potential to change the standard
of care for the patient population suffering from APDS."
Vicki Modell, co-founder of
the Jeffrey Modell Foundation, an international, non-profit,
organization dedicated to helping individuals and family members
affected by primary immunodeficiency disorders,
commented:
"The approval of Pharming's Joenja® is an
important step toward making a difference in the lives of
individuals living with APDS who experience severe, life-altering
and progressive symptoms. The FDA approval of a treatment option
for one of the more than 450 primary immunodeficiencies is also a
key moment for the broader primary immunodeficiency community. The
Jeffrey Modell Foundation's mission of hope, advocacy and action is
dedicated to early diagnosis, genetic sequencing, treatments and
ultimately, future cures for primary immunodeficiencies."
Sijmen de Vries, Chief Executive Officer of Pharming,
commented:
"This FDA approval of Joenja® is an important milestone for
people living with APDS who will now have access to the first
approved treatment option specifically for this debilitating
disease. Until now, management of APDS has relied on the treatment
of the diverse symptoms associated with APDS. We are grateful to
the patients, caregivers, and physicians who participated in the
clinical trials who have made today's approval a reality. I would
also like to thank the Pharming and the Novartis teams who have
supported the development of Joenja® and can, therefore, be
justifiably proud of this FDA approval. Today also marks a landmark
event for Pharming and demonstrates our commitment to transforming
the lives of patients who suffer from rare diseases. The approval
and near-term launch of Joenja®, our second commercial product,
brings us closer to our goal of becoming a leading global rare
disease company dedicated to patient communities with unmet
medical needs."
APDS is a rare primary immunodeficiency that was first
characterized in 2013 and is currently estimated to affect 1 to 2
people per million. It is caused by genetic variants in either one
of two identified genes, known as PIK3CD or PIK3R1,
which are vital to the normal development and function of immune
cells in the body. While people with APDS may suffer from a wide
variety of symptoms, the most common are frequent and severe
infections of the ears, sinuses, and upper and lower respiratory
tracts. Infections usually begin in infancy. People with APDS are
susceptible to swollen lymph nodes or an enlarged spleen
(splenomegaly), as well as autoimmunity and inflammatory symptoms.
People with APDS may also be at higher risk for cancers like
lymphoma.
The FDA evaluated the New Drug Application (NDA) for Joenja®
under priority review and has approved the drug based on findings
from a multinational, triple-blind, placebo-controlled, randomized
Phase II/III clinical trial, which 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 38 patients received
Joenja® for a median of two years.
Results from the 12-week randomized, placebo-controlled study in
31 patients with APDS aged 12 years and older demonstrated clinical
efficacy of Joenja® 70mg twice daily over placebo, and was
significant in the co-primary endpoints which evaluated improvement
in lymphoproliferation as measured by the reduction in lymph node
size and increase in naïve B cells, reflecting the impact on immune
dysregulation and normalization of immunophenotype in these
patients, respectively. The adjusted mean change (95% CI) between
Joenja® and placebo for lymph node size was -0.25 (–0.38,-0.12;
P=0.0006; N=26) and for percentage of naïve B cells was 37.30
(24.06, 50.54; P=0.0002; N=13). The most common adverse reactions
in the clinical trial (incidence >10%) were headache, sinusitis,
and atopic dermatitis.
With the approval of Joenja®, as a treatment for a rare
pediatric disease, the FDA granted Pharming a priority review
voucher ("PRV"). Pursuant to the terms of Pharming's 2019
exclusive license agreement with Novartis for leniolisib, Novartis
has the right to purchase the PRV from Pharming for a small
minority share of the value of the PRV. Pursuant to the agreement,
Pharming will make milestone payments to Novartis and another party
for the approval and first commercial sale for APDS totaling
$10.5 million and we agreed to make
certain additional milestone payments to Novartis in an aggregate
amount of up to $190 million upon the
achievement of certain leniolisib sales milestones. We also agreed
to make tiered royalty payments to Novartis, calculated as low
double-digit to high-teen double-digit percentage of net sales of
leniolisib.
For more information regarding APDS, please visit
AllAboutAPDS.com. Pharming, in collaboration with Invitae
Corporation, facilitates access to no charge genetic testing and
counseling in the US and Canada
through NavigateAPDS.com. For more information regarding Pharming
and their dedication to the rare disease community, please visit
www.Pharming.com.
The Marketing Authorisation Application (MAA) for leniolisib
with the European Medicines Agency's (EMA) Committee for Human
Medicinal Products (CHMP) is currently under review. Pharming
expects that CHMP will issue its opinion on the MAA in the second
half of 2023.
US Important Safety Information for
Joenja® (leniolisib)
INDICATIONS AND USAGE
Joenja® (leniolisib) is a kinase inhibitor indicated for the
treatment of activated phosphoinositide 3-kinase delta (PI3Kδ)
syndrome (APDS) in adult and pediatric patients 12 years of age and
older.
IMPORTANT SAFETY INFORMATION
Verify pregnancy status in females of reproductive potential
prior to initiating treatment with Joenja®.
Joenja® may cause fetal harm when administered to a pregnant
woman. Advise patients of the potential risk to a fetus and to use
highly effective methods of contraception during treatment with
Joenja® and for 1 week after the last dose of Joenja®.
Live, attenuated vaccinations may be less effective if
administered during Joenja® treatment.
Use of Joenja® in patients with moderate to severe hepatic
impairment is not recommended. There is no recommended dosage for
patients weighing less than 45 kg.
The most common adverse reactions (incidence >10%) seen in
clinical trials were headache, sinusitis, and atopic
dermatitis.
Seven (33%) patients receiving Joenja® developed an absolute
neutrophil count (ANC) between 500 and 1500 cells/microL. No
patients developed an ANC <500 cells/microL and there were no
reports of infection associated with neutropenia.
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
dysregulation.1,2,3 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 Joenja® (leniolisib)
Joenja® (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. Joenja® 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 Joenja® in the co-primary
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 Joenja®
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. For information about Joenja®, visit:
Joenja.com
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 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.
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(1):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.
8. RAO VK, et al Blood. 2023 Mar
2;141(9):971-983.
Investors and Analysts
conference call dial-in information
March 27, 2023: 14:00CEST/08:00EST
Please note, the Company will only take questions from dial-in
attendees.
Dial-in details:
Netherlands
(Local)
+31 85 888 7233
United
Kingdom
+44 808 189 0158
United Kingdom
(Local)
+44 20 3936 2999
United
States
+1 855 979 6654
United States
(Local)
+1 646 664 1960
Global Dial-In Numbers
Access code: 040991
Webcast
link: https://webcast.openbriefing.com/pharming-mar23/
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
EU PR
Claire Dobbs
E: claire.dobbs@solarishealth.com
T: +44 7864 640093
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