- Positive opinion based on Phase 3 ADAPT trial showing
efgartigimod provided clinically meaningful improvements in
strength and quality of life measures
- European Commission decision on marketing authorization
application (MAA) expected in approximately 60 days
- If approved, efgartigimod will be the first neonatal Fc
receptor (FcRn) blocker for the treatment of adults in Europe
living with rare neuromuscular disease generalized myasthenia
gravis (gMG)
Breda, the
Netherlands—June
24, 2022—argenx
(Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases, today announced that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has recommended European Commission (EC) approval of
efgartigimod as an add-on to standard therapy for the treatment of
adult patients with generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AChR) antibody positive.
The positive CHMP opinion is a scientific
recommendation for marketing authorization, serving as a basis for
the EC’s final decision on argenx’s application for efgartigimod.
The EC is expected to make a decision within approximately 60 days
following CHMP recommendation, which will be applicable to all 27
European Union Member States, in addition to Iceland, Norway and
Liechtenstein.
“The current treatment of generalized MG in
Europe leaves many patients with insufficiently controlled symptoms
that markedly decrease quality of life. Caregivers and medical
teams are well aware of the need for new treatment options that are
safe, effective and targeted to the disease biology” said Anthony
Béhin, MD neuromuscular physician at Institut de Myologie, La Pitié
Salpétrière, Paris. “As a practicing neurologist regularly seeing
people who live with this debilitating chronic disease, the CHMP’s
positive opinion of efgartigimod represents an exciting advancement
toward bringing a new treatment option to these patients in
Europe.”
The MAA included results from the pivotal Phase
3 ADAPT trial, which were published in the July 2021 issue of The
Lancet Neurology. The ADAPT trial met its primary endpoint,
demonstrating that significantly more anti-acetylcholine receptor
(AChR) antibody positive gMG patients were responders on the
Myasthenia Gravis Activities of Daily Living (MG-ADL) scale
following treatment with efgartigimod compared with placebo (68%
vs. 30%; p<0.0001). Responders were defined as having at least a
two-point reduction on the MG-ADL scale sustained for four or more
consecutive weeks during the first treatment cycle.
There were additionally significantly more
responders on the Quantitative Myasthenia Gravis (QMG) scale
following treatment with efgartigimod compared with placebo (63%
vs. 14%; p<0.0001). Responders were defined as having at least a
three-point reduction on the QMG scale sustained for four or more
consecutive weeks during the first treatment cycle.
Efgartigimod had a demonstrated safety profile in the ADAPT
clinical trial. The most frequently reported adverse reactions were
upper respiratory tract infections (10.7% following treatment with
efgartigimod vs. 4.8% of placebo) and urinary tract infections
(9.5% vs. 4.8%).
“We are thrilled by the CHMP’s recommendation in
favor of efgartigimod, which brings us one step closer to
delivering this therapy to people living with gMG in Europe and
around the world,” said Anant Murthy, Ph.D., General Manager, EU,
argenx. “We are confident in the European team we have built, and
pending marketing authorization, look forward to close
collaboration with regulatory bodies and government authorities
across the region to ensure this treatment option will be available
for as many patients as possible.”
Efgartigimod is the first-and-only approved FcRn
blocker in the U.S. as VYVGART (efgartigimod alfa-fcab) for the
treatment of adult gMG patients who are anti-AChR antibody positive
and in Japan for those who do not have sufficient response to
steroids or non-steroidal immunosuppressive therapies (ISTs).
argenx also plans to launch efgartigimod in Canada, China through
its collaboration with Zai Lab, and select additional regions.
About Phase 3 ADAPT Trial
The Phase 3 ADAPT trial was a 26-week
randomized, double-blind, placebo-controlled, multi-center, global
trial evaluating the safety and efficacy of efgartigimod in adult
patients with gMG. A total of 167 adult patients with gMG in North
America, Europe and Japan enrolled in the trial. Patients were
randomized in a 1:1 ratio to receive efgartigimod or placebo, in
addition to stable doses of their current gMG treatment. ADAPT was
designed to enable an individualized treatment approach with an
initial treatment cycle followed by subsequent treatment cycles
based on clinical evaluation. The primary endpoint was the
comparison of percentage of MG-ADL responders in the first
treatment cycle between efgartigimod and placebo treatment groups
in the anti-AChR antibody positive population.
About Efgartigimod
Efgartigimod is an antibody fragment designed to
reduce pathogenic immunoglobulin G (IgG) antibodies by binding to
the neonatal Fc receptor and blocking the IgG recycling process.
Efgartigimod is being investigated in several autoimmune diseases
known to be mediated by disease-causing IgG antibodies, including
neuromuscular disorders, blood disorders, and skin blistering
diseases. It is currently approved in the United States for the
treatment of adult patients with gMG who are anti-acetylcholine
receptor antibody positive, and Japan for adult patients with gMG
who do not have sufficient response to steroids or non-steroidal
immunosuppressive therapies.
About Generalized Myasthenia
Gravis
Generalized myasthenia gravis (gMG) is a rare
and chronic autoimmune disease where IgG autoantibodies disrupt
communication between nerves and muscles, causing debilitating and
potentially life-threatening muscle weakness. Approximately 85% of
people with MG progress to gMG within 24 months1, where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population1.
About argenx
argenx is a global immunology company committed
to improving the lives of people suffering from severe autoimmune
diseases. Partnering with leading academic researchers through its
Immunology Innovation Program (IIP), argenx aims to translate
immunology breakthroughs into a world-class portfolio of novel
antibody-based medicines. argenx developed and is commercializing
the first-and-only approved neonatal Fc receptor (FcRn) blocker in
the U.S. and Japan.
For further information, please contact:
Media:Kelsey Kirkkkirk@argenx.com
Investors:Beth
DelGiaccobdelgiacco@argenx.com
Michelle Greenblattmgreenblatt@argenx.com
Forward-looking Statements The contents of this
announcement include statements that are, or may be deemed to be,
“forward-looking statements.” These forward-looking statements can
be identified by the use of forward-looking terminology, including
the terms “believes,” “hope,” “estimates,” “anticipates,”
“expects,” “intends,” “may,” “will,” or “should” and include
statements argenx makes concerning the timing of any approval or
marketing authorization by the EC of efgartigimod as an add-on to
standard therapy for the treatment of adult patients with gMG who
are AChR antibody positive. By their nature, forward-looking
statements involve risks and uncertainties and readers are
cautioned that any such forward-looking statements are not
guarantees of future performance. argenx’s actual results may
differ materially from those predicted by the forward-looking
statements as a result of various important factors. A further list
and description of these risks, uncertainties and other risks can
be found in argenx’s U.S. Securities and Exchange Commission (SEC)
filings and reports, including in argenx’s most recent annual
report on Form 20-F filed with the SEC as well as subsequent
filings and reports filed by argenx with the SEC. Given these
uncertainties, the reader is advised not to place any undue
reliance on such forward-looking statements. These forward-looking
statements speak only as of the date of publication of this
document. argenx undertakes no obligation publicly update or revise
the information in this press release, including any
forward-looking statements, except as may be required by law.
1 Behin et al. New Pathways and Therapeutics Targets in
Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277
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