Full Press Release Details
argenx Receives Positive
CHMP Opinion for Efgartigimod for the Treatment of Adult Patients with Generalized Myasthenia Gravis in Europe
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
B hin, MD neuromuscular physician at Institut de Myologie, La Piti Salp tri re, 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.
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.
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.
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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
1 Behin et al. New Pathways and Therapeutics Targets in Autoimmune
Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277