argenx Announces Validation of European Marketing Authorization Application for Efgartigimod in Generalized Myasthenia Gravis
25 Août 2021 - 7:00AM
- Validation initiates formal evaluation of application which
started on August 19, 2021; decision expected mid-2022
- If approved, efgartigimod will be the first-and-only approved
FcRn antagonist in Europe
- Regulatory reviews of efgartigimod for generalized myasthenia
gravis currently underway in the U.S., Japan and Europe
Breda, the Netherlands –
August
25, 2021 –
argenx (Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases and cancer, today announced that it has
submitted, and the European Medicines Agency (EMA) has validated,
the marketing authorization application (MAA) for the Company’s
investigational FcRn antagonist, efgartigimod, for the treatment of
generalized myasthenia gravis (gMG). Validation of the MAA confirms
that the application is sufficiently complete to begin the formal
review process.
The MAA is supported by results from the pivotal
Phase 3 ADAPT trial evaluating the safety and efficacy of
efgartigimod for the treatment of patients with gMG.
“gMG is a severe, chronic and debilitating
disease that can be unpredictable and greatly impact a person’s
quality of life. The EMA’s validation is an exciting step closer to
our goal of helping people globally who are living with this
disease in which there remains a significant unmet need.” said Tim
Van Hauwermeiren, Chief Executive Officer of argenx. “We have been
building an experienced commercial team in Europe and look forward
to our continued collaboration with European regulatory authorities
through the review process.”
“There remains a significant unmet need for new
gMG treatment options that are targeted to the underlying
pathogenesis of the disease and supported by clinical data,”
commented Prof. Dr. Andreas Meisel, Senior Physician of Neurology,
Head of Myasthenia Gravis Outpatient Clinic at the Department of
Neurology, Charité – Universitätsmedizin Berlin, and Principal
Investigator on the ADAPT study. “Myasthenia gravis can severely
impact a person’s quality of life and ability to carry out daily
tasks, such as speaking, chewing and swallowing food, or brushing
teeth and hair. In some cases, patients may also experience
difficulty breathing. I am hopeful for continued research
advancements for new options to treat this debilitating
disease.”
Efgartigimod is currently under review with the
U.S. Food and Drug Administration (FDA) for the treatment of gMG
with a Prescription Drug User Fee Act (PDUFA) target action date of
December 17, 2021. argenx also submitted and was notified of
acceptance of an application for efgartigimod to Japan’s
Pharmaceuticals and Medical Devices Agency (PMDA) earlier this
year.
Phase 3 ADAPT Trial
The Phase 3 ADAPT trial was a randomized,
double-blind, placebo-controlled, multi-center, global trial
evaluating the safety and efficacy of efgartigimod in patients with
gMG. A total of 167 adult patients with gMG in North America,
Europe and Japan enrolled in the trial and were treated. Patients
were eligible to enroll in ADAPT regardless of antibody status,
including patients with acetylcholine receptor (AChR) antibodies
(AChR-Ab+) and patients where AChR antibodies were not detected.
Patients were randomized in a 1:1 ratio to receive efgartigimod or
placebo for a total of 26 weeks. ADAPT was designed to enable an
individualized treatment approach with an initial treatment cycle
followed by a variable number of subsequent treatment cycles. The
primary endpoint was the number of AChR-Ab+ patients who achieved a
response on the MG-ADL score defined by at least a two-point
improvement for four or more consecutive weeks.
About Efgartigimod
Efgartigimod is an investigational 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.
About Myasthenia Gravis
Myasthenia gravis (MG) is a rare and chronic
autoimmune disease, often causing debilitating and potentially
life-threatening muscle weakness. More than 85% of people with MG
progress to generalized MG (gMG) within 18 months, where muscles
throughout the body may be affected, resulting in extreme fatigue
and difficulties with facial expression, speech, swallowing and
mobility. In more life-threatening cases, MG can affect the muscles
responsible for breathing.
About argenx
argenx is a global immunology company committed
to improving the lives of people suffering from severe autoimmune
diseases and cancer. 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 is evaluating efgartigimod
in multiple serious autoimmune diseases. argenx is also advancing
several earlier stage experimental medicines within its therapeutic
franchises. argenx has offices in Belgium, the United States,
Japan, and Switzerland. For more information, visit www.argenx.com
and follow us on LinkedIn.
Media:Kelsey Kirkkkirk@argenx.com
Joke Comijn (EU)jcomijn@argenx.com
Investors:Beth
DelGiaccobdelgiacco@argenx.com
Michelle Greenblattmgreenblatt@argenx.com
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, estimates, anticipates, expects, intends, may, will, or
should, and include statements argenx makes concerning: its
expected collaboration with European regulatory authorities; its
statements regarding therapeutic potential of efgartigimod in
patients; the commercial potential of efgartigimod; its clinical
development and regulatory plans, including the timing and outcome
of regulatory filings and approvals, including those with FDA, PMDA
and EMA described in this announcement and the timing and progress
of commercialization activities; and its statements regarding
research advancements and number of treatment options. 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, including the
effects of the COVID-19 pandemic, the inherent uncertainties
associated with preclinical and clinical trial and product
development activities and regulatory approval requirements;
argenx’s reliance on collaborations with third parties; estimating
the commercial potential of argenx’s product candidates; argenx’s
ability to obtain and maintain protection of intellectual property
for its technologies and drugs; argenx’s limited operating history;
and argenx’s ability to obtain additional funding for operations
and to complete the development and commercialization of its
product candidates. 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 by law.
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