Full Press Release Details
argenx announces VYVGART approval in
Japan for the treatment of generalized myasthenia gravis
Regulated Information/Inside
Breda, the Netherlands - Jan. 20, 2022
- argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering
from severe autoimmune diseases, today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved VYVGART
(efgartigimod alfa) intravenous infusion for the treatment of adult patients with generalized myasthenia gravis (gMG) who do not have
sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). VYVGART is the first-and-only neonatal Fc receptor
(FcRn) blocker approved in Japan.
"People living with gMG around the world
continue to experience severe disease burden despite treatment with commonly-used therapies. We are extremely proud to deliver the first-and-only
approved FcRn blocker in Japan to a broad population of gMG patients, regardless of antibody status," said Tim Van Hauwermeiren,
Chief Executive Officer of argenx. "Our commercial teams are ready and motivated to be serving as many people as possible who are
living with this debilitating disease and we look forward to collaborating with the Japanese government to enable patient access. With
today's approval of VYVGART in Japan, the recent U.S. FDA approval, and ongoing review of our application in Europe, we continue
to advance rapidly toward achieving our goal of delivering this innovative, targeted treatment option globally."
Generalized myasthenia gravis is a rare and chronic neuromuscular disease
characterized by debilitating and potentially life-threatening muscle weakness. VYVGART is a human IgG1 antibody fragment that binds to
FcRn, resulting in the reduction of circulating immunoglobulin G (IgG) autoantibodies. The action of IgG autoantibodies at the neuromuscular
junction is a key driver of gMG.²
The approval of VYVGART is based on results from
the global 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 VYVGART 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 also significantly more responders
on the Quantitative Myasthenia Gravis (QMG) scale following treatment with VYVGART 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
VYVGART had a demonstrated safety profile in the
ADAPT clinical trial. The most common adverse events in ADAPT were respiratory tract infection (33% vs. 29% placebo), headache (32% vs.
29% placebo), and urinary tract infection (10% vs. 5% placebo).
VYVGART was approved by the U.S. Food and Drug Administration (FDA)
on December 17, 2021 for the treatment of gMG in adult patients who are AChR antibody positive. A Marketing Authorization Application
for efgartigimod for the treatment of gMG is currently under review by the European Medicines Agency (EMA), with a decision anticipated
in the second half of 2022. argenx is evaluating efgartigimod in six high-need autoimmune conditions, set to expand to ten conditions
See the full Prescribing Information for VYVGART
in the U.S., which includes the below Important Safety Information. For more information related to VYVGART in Japan, visit argenx.jp.
Safety Information (U.S. prescribing information)
What is VYVGARTTM (efgartigimod
VYVGART is a prescription medicine used to treat
a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are
positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).
What is the most important information I should
VYVGART may cause serious side effects, including:
Before taking VYVGART, tell your health care provider
about all of your medical conditions, including if you:
Tell your health care provider about all the medicines
you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the common side effects of VYVGART?
The most common side effects of VYVGART are respiratory tract infection,
headache, and urinary tract infection.
These are not all the possible side effects of
VYVGART. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at
Please see the full Prescribing Information for
VYVGART and talk to your doctor.
VYVGART (efgartigimod alfa-fcab) is a human
IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating immunoglobulin G (IgG)
autoantibodies. It is the first and only approved FcRn blocker. VYVGART is approved in the United States for the treatment of adults with
generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults
with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).
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 population.1
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. The Company
is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its
Twitter, and Instagram.
1 Behin et al. New Pathways and Therapeutics
Targets in Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277
2 Howard JF Jr, Utsugisawa K, Benatar
M, et al. Safety and efficacy of efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia
gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicenter study. Lancet Neurol. 2017; 16: 976-86
For further information, please contact:
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 its global launch strategy; its expectation concerning treatment options, scale of potential patients and impact and
effect on patients; estimates concerning the commercialization potential of VYVGART; anticipated outcome of collaboration with the Japanese
government; expected approval of VYVGART by European Medicines Agency of MAA in the second half of 2022; and evaluation of efgartigimod
in up to ten high-need conditions by end of 2022. 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
to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required