-
VYVGART® is first-and-only targeted IgG
Fc-antibody fragment for CIDP
-
First novel mechanism of action for CIDP treatment in more than 30
years
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CHMP positive opinion based on ADHERE data, the largest ever CIDP
clinical trial
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European Commission (EC) decision on marketing authorization
application (MAA) expected within approximately two
months
April 28, 2025, 07:00 AM CET
Amsterdam, the Netherlands – argenx SE
(Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases, today announced the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency
(EMA) has recommended European Commission (EC) approval of VYVGART®
1000mg (efgartigimod alfa) for subcutaneous (SC) injection as a
monotherapy for the treatment of adult patients with progressive or
relapsing active chronic inflammatory demyelinating polyneuropathy
(CIDP) after prior treatment with corticosteroids or
immunoglobulins.
“Our mission is to develop innovative, targeted
treatments for patients with rare and severe autoimmune diseases,
who continue to face significant unmet needs. The positive CHMP
opinion for VYVGART in CIDP brings us one step closer to providing
patients across Europe with a transformational new treatment option
that provides meaningful functional improvement,” said Luc Truyen
M.D., Ph.D., Chief Medical Officer, argenx. “VYVGART is the first
and only targeted IgG Fc-antibody fragment for CIDP and if
approved, would mark the first treatment in Europe with a novel,
precision mechanism of action for CIDP patients in 30 years.”
VYVGART for subcutaneous injection is available
as a vial or prefilled syringe and can be administered by a
patient, caregiver, or healthcare professional. Treatment is
initiated with a weekly dose regimen and may be adjusted to every
other week based on clinical evaluation.
The CHMP recommendation is based on positive
results from the ADHERE clinical trial, the largest study of CIDP
patients to date. In the ADHERE study, 66.5% (214/322) of patients
treated with VYVGART, regardless of prior treatment, demonstrated
evidence of clinical improvement, including improvements in
mobility, function and strength. ADHERE met its primary endpoint
(p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25;
0.61) in the risk of relapse versus placebo. The study also
demonstrated functional improvements across the Inflammatory
Neuropathy Cause and Treatment (INCAT) disability scores
(>1-point), grip strength (>17 kPa) and I-RODS scale (>8
points) at week 36 compared to baseline at entry to standard of
care withdrawal phase. Ninety-nine percent of trial participants
elected to participate in the ADHERE open-label extension. The
safety results were generally consistent with the known safety
profile of VYVGART in previous clinical studies.
"For the patient population represented by the
European Patient Organisation for Dysimmune and Inflammatory
Neuropathies (EPODIN) and for those affected by CIDP, this is
excellent news," said Jean-Philippe Plançon, President of EPODIN.
"There are still considerable unmet medical needs in the management
of CIDP, and the CHMP’s recommendation brings renewed hope for
improved treatment options and quality of life."
The positive CHMP opinion is a scientific
recommendation for marketing authorization, serving as a basis for
the EC’s final decision on argenx’s CIDP application for
subcutaneous VYVGART. The EC is expected to make a decision
following CHMP recommendation and the decision will apply to all 27
European Union Member States, and also to Iceland, Norway and
Liechtenstein. Currently, VYVGART is indicated 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.
About ADHERE
The ADHERE trial was a multicenter, randomized,
double-blind, placebo-controlled trial evaluating SC efgartigimod
alfa for the treatment of chronic inflammatory demyelinating
polyneuropathy (CIDP). ADHERE enrolled 322 adult patients with
CIDP, 130 of whom were based in Europe, who were off treatment (not
on active treatment within the past six months or newly diagnosed)
or being treated with immunoglobulin therapy or corticosteroids.
The trial consisted of an open-label Stage A followed by a
randomized, placebo-controlled Stage B. In order to be eligible for
the trial, the diagnosis of CIDP was confirmed by an independent
panel of experts. Patients entered a run-in stage, where any
ongoing CIDP treatment was stopped and in order to be eligible for
Stage A had to demonstrate active disease, with clinically
meaningful worsening on at least one CIDP clinical assessment tool,
including INCAT, I-RODS, or mean grip strength. Treatment naïve
patients were able to skip the run-in period with proof of recent
worsening. To advance to Stage B, patients needed to demonstrate
evidence of clinical improvement (ECI) with SC efgartigimod alfa.
ECI was achieved through improvement of the INCAT score, or
improvement on I- RODS or mean grip strength if those scales had
demonstrated worsening during the run-in period. In Stage B,
patients were randomized to either SC efgartigimod alfa or placebo
for up to 48 weeks. The primary endpoint was measured once 88 total
relapses or events were achieved in Stage B and was based on the
hazard ratio for the time to first adjusted INCAT deterioration
(i.e. relapse). After Stage B, all patients had the option to
roll-over to an open-label extension study to receive SC
efgartigimod alfa.
About Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP)Chronic inflammatory demyelinating
polyneuropathy (CIDP) is a rare and serious autoimmune disease of
the peripheral nervous system. There is increasing evidence that
IgG antibodies play a key role in the damage to the peripheral
nerves. People with CIDP experience fatigue, muscle weakness and a
loss of feeling in their arms and legs that can worsen over time or
may come and go. These symptoms can significantly impair a person's
ability to function in their daily lives. Without treatment,
one-third of people living with CIDP will need a wheelchair. There
are an estimated 31,413 people living with CIDP in the European
Union.
About EfgartigimodEfgartigimod
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 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 approved neonatal Fc receptor (FcRn) blocker and is
evaluating its broad potential in multiple serious autoimmune
diseases while advancing several earlier stage experimental
medicines within its therapeutic franchises. For more information,
visit www.argenx.com and follow us on LinkedIn, Instagram,
Facebook, and YouTube.
Contacts
Media:Kate Dion kdion@argenx.com
Investors: Alexandra
Royaroy@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 “aim,”
“are,” “believe,” “can,” “continue,” “expect,” “may,” and “will”
and include statements argenx makes concerning the expected timing
and decision of the EC regarding VYVGART for SC injection for CIDP
treatment and the application of such decision; the potential for
improved treatment options and quality of life; and its goal of
translating immunology breakthroughs into a world-class portfolio
of novel antibody-based medicines. 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 but
not limited to, the results of argenx’s clinical trials;
expectations regarding the inherent uncertainties associated with
the development of novel drug therapies; preclinical and clinical
trial and product development activities and regulatory approval
requirements; the acceptance of its products and product candidates
by its patients as safe, effective and cost-effective; the impact
of governmental laws and regulations, including tariffs, export
controls, sanctions and other regulations on its business; its
reliance on third-party suppliers, service providers and
manufacturers; inflation and deflation and the corresponding
fluctuations in interest rates; and regional instability and
conflicts. 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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