- Positive
opinion based on Phase 3 ADAPT-SC study demonstrating noninferior
total IgG reduction at day 29 with subcutaneously (SC) administered
efgartigimod, compared to intravenous (IV) administration
- European
Commission (EC) decision on marketing authorization application
(MAA) expected within approximately 60 days
Amsterdam, The
Netherlands—September
15, 2023—argenx
SE (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 EC approval of the SC injectable
formulation 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.
SC efgartigimod is formulated with Halozyme’s ENHANZE® drug
delivery technology to facilitate subcutaneous delivery of
biologics.
“Generalized myasthenia gravis is a complex and
devastating disease that is debilitating for people who live with
it, making routine movements exhausting and challenging to
perform,” said Prof. dr. Jan De Bleecker, Ghent University Hospital
and Ghent University. “I’m pleased to learn of the CHMP’s positive
opinion as it represents a significant advancement for the gMG
community who would benefit from an additional, effective treatment
option that can improve quality of life and better manage this
chronic condition. In particular, SC efgartigimod has the potential
to have a positive impact on treatment convenience, leading to a
broader positive impact for patients and healthcare systems.”
“The positive recommendation by the CHMP for the
SC injectable formulation of efgartigimod brings us one step closer
to broadening our treatment offering for people living with gMG in
Europe,” said Anant Murthy, Ph.D., General Manager, EMEA, argenx.
“Our mission is to transform the treatment of severe autoimmune
disease, and we remain committed to providing gMG patients a second
innovation that could further address treatment burden. We are
particularly pleased with the possibility for self-administration
of the SC formulation, which may provide additional treatment
flexibility for physicians and patients.”
The CHMP recommendation is based on positive
results from the Phase 3 ADAPT-SC study. The ADAPT-SC trial met its
primary endpoint of noninferiority, where SC efgartigimod
demonstrated a mean total IgG reduction of 66.4% from baseline at
day 29, compared to 62.2% with the IV formulation. Additional key
secondary endpoints were met, which were consistent with efficacy
measures from the ADAPT IV study identifying the correlation
between IgG reduction and clinical benefit in gMG.
SC efgartigimod has a demonstrated safety
profile, consistent with the ADAPT IV clinical trial with the
exception of injection site reactions (ISRs), which are commonly
observed with biologics administered subcutaneously. ISRs were mild
to moderate and did not lead to treatment discontinuation.
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 SC
efgartigimod. The EC is expected to make a decision within
approximately 60 days following CHMP recommendation. The decision
will apply to all 27 European Union Member States, and also to
Iceland, Norway and Liechtenstein.
About Phase 3 ADAPT-SC
Trial
The Phase 3 ADAPT-SC trial was a multicenter,
randomized, open-label, parallel-group study evaluating the
noninferiority of the pharmacodynamic (PD) effect of SC
efgartigimod compared with IV efgartigimod in adult patients with
gMG. The PD effect was measured by percent change from baseline in
autoantibody (AChR) levels at day 29. Safety, clinical efficacy,
immunogenicity and pharmacokinetics (PK) were also assessed. A
total of 110 adult patients with gMG in North America, Europe and
Japan enrolled in the ADAPT-SC trial. Patients were randomized in a
1:1 ratio to receive SC efgartigimod for one treatment cycle
consisting of four doses at once-weekly intervals. The total study
duration was approximately 12 weeks, including seven weeks of
follow-up after the treatment cycle. At the completion of ADAPT-SC,
patients had the opportunity to roll-over to ADAPT-SC+, an
open-label extension study.
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, in both an IV and SC formulation. SC efgartigimod is
co-formulated with recombinant human hyaluronidase PH20 (rHuPH20),
Halozyme's ENHANZE® drug delivery technology. In August 2022,
efgartigimod received approval from the EC for IV administration as
an add on to standard therapy for the treatment of adult patients
with gMG who are AChR antibody positive.
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 months, where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population.
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., Japan, Israel, the EU and the UK. The Company is
evaluating efgartigimod in multiple serious autoimmune diseases and
advancing several earlier stage experimental medicines within its
therapeutic franchises.
For further information, please contact:
Media:
Erin MurphyEMurphy@argenx.com
Investors:
Alexandra Roy (US)ARoy@argenx.com
Lynn Elton (EU)LElton@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 approval or marketing authorization by the EC of the
SC injectable formulation of efgartigimod as an add on to standard
therapy for the treatment of adult patients with gMG who are AChR
antibody positive; and the benefits and impact of SC efgartigimod
on patients, physicians and healthcare systems. 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 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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