VYVDURA® now approved for
at-home self-injection in Japan for both generalized myasthenia
gravis and CIDP
argenx's VYVGART® and VYVDURA
portfolio approved in Japan for three indications – first country
globally with access across three indications
December 27, 2024, 7:00 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 that Japan’s Ministry
of Health, Labour and Welfare (MHLW) approved VYVDURA for adults
with chronic inflammatory demyelinating polyneuropathy (CIDP).
VYVDURA is approved for CIDP as a once weekly 30-to-90 second
subcutaneous injection, which can be self-administered at home, and
is the first and only neonatal Fc receptor (FcRn) blocker approved
for the treatment of CIDP.
“CIDP is a rare and debilitating disease for
which there has been little innovation in treatment in 30 years,”
said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx.
“With VYVDURA, CIDP patients in Japan now have access to a novel
therapy with a focused mode of action offering a convenient
30-to-90 second at-home self-injection option with an established
efficacy and safety profile, as demonstrated by the ADHERE trial
and real-world evidence. By extending the reach of this
transformational therapy to thousands more patients, argenx
continues to bring efgartigimod, our first-in-class FcRn blocker,
to more patients in Japan and around the world suffering from
severe autoimmune disease.”
CIDP is a progressive, immune-mediated rare and
debilitating neuromuscular disorder of the peripheral nervous
system. Patients experience a range of disabling mobility and
sensory issues, including trouble standing from a seated position,
pain and fatigue, and frequent tripping or falling. Many patients
become wheelchair bound and are unable to work as the disease
progresses. Currently, 85% of patients require ongoing treatment
and nearly 88% of treated patients experience residual impairment
and disability.
The MHLW approval is based on the
ADHERE Study, the largest clinical trial to date studying
CIDP. In the ADHERE study, 69% (221/322) of patients treated with
VYVDURA, 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. 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 VYVDURA in previous clinical studies and
real-world use.
VYVDURA was approved by the MHLW for
manufacturing and marketing in January 2024 and launched in April
2024 for the treatment of generalized myasthenia gravis (gMG). In
March 2024, VYVDURA was designated as an Orphan Drug for the
treatment of CIDP by the MHLW.
See FDA-approved Important Safety Information
below and full Prescribing Information for VYVDURA, which
is marketed as VYVGART Hytrulo in the United States, for additional
information.
What is
VYVGART® HYTRULO (efgartigimod alfa and
hyaluronidase-qvfc)?VYVGART HYTRULO is a prescription
medicine used for the treatment of adult patients with chronic
inflammatory demyelinating polyneuropathy (CIDP).
IMPORTANT
SAFETY INFORMATIONDo not use
VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa,
hyaluronidase, or any of the other ingredients in VYVGART HYTRULO.
VYVGART HYTRULO can cause serious allergic reactions and a decrease
in blood pressure leading to fainting.
VYVGART
HYTRULO may
cause serious
side effects,
including:
- Infection. VYVGART
HYTRULO may increase the risk of infection. The most common
infections for efgartigimod alfa-fcab-treated patients were urinary
tract and respiratory tract infections. Signs or symptoms of an
infection may include fever, chills, frequent and/or painful
urination, cough, pain and blockage of nasal passages/sinus,
wheezing, shortness of breath, fatigue, sore throat, excess phlegm,
nasal discharge, back pain, and/or chest pain.
- Allergic Reactions
(hypersensitivity reactions). VYVGART HYTRULO can cause
allergic reactions such as rashes, swelling under the skin, and
shortness of breath. Hives were also observed in patients treated
with VYVGART HYTRULO. Serious allergic reactions, such as trouble
breathing and decrease in blood pressure leading to fainting have
been reported with efgartigimod alfa-fcab.
- Infusion-Related
Reactions. VYVGART HYTRULO can cause infusion-related
reactions. The most frequent symptoms and signs reported with
efgartigimod alfa-fcab were high blood pressure, chills, shivering,
and chest, abdominal, and back pain.
Tell your doctor if you have signs or symptoms
of an infection, allergic reaction, or infusion-related reaction.
These can happen while you are receiving your VYVGART HYTRULO
treatment or afterward. Your doctor may need to pause or stop your
treatment. Contact your doctor immediately if you have signs or
symptoms of a serious allergic reaction.
Before taking VYVGART HYTRULO, tell your
doctor if you:
- take any medicines, including
prescription and non-prescription medicines, supplements, or herbal
medicines,
- have received or are scheduled to
receive a vaccine (immunization), or
- have any allergies or medical
conditions, including if you are pregnant or planning to become
pregnant, or are breastfeeding.
What are
the common side
effects of VYVGART
HYTRULO?The most common side effects in
efgartigimod-alfa-fcab-treated patients were respiratory tract
infection, headache, and urinary tract infection. Additional common
side effects with VYVGART HYTRULO are injection site reactions,
including rash, redness of the skin, itching sensation, bruising,
pain, and hives.
These are not all the possible side effects of
VYVGART HYTRULO. Call your doctor for medical advice about side
effects. You may report side effects to the US Food and Drug
Administration at 1-800-FDA-1088.
Please see
the full
Prescribing Information
for VYVGART HYTRULO and talk to your
doctor.
About ADHERE Trial Design
The ADHERE trial was a multicenter, randomized,
double-blind, placebo-controlled trial evaluating VYVDURA®
(efgartigimod alfa and hyaluronidase-qvfc) for the treatment of
chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE
enrolled 322 adult patients with CIDP who were treatment naïve (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 VYVDURA. 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 VYVDURA 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 VYVDURA.
About VYVDURA
VYVDURA is a subcutaneous combination of
efgartigimod alfa, a human IgG1 antibody fragment marketed for
intravenous use as VYVGART, and recombinant human hyaluronidase
PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology
to facilitate subcutaneous injection delivery of biologics. In
binding to the neonatal Fc receptor (FcRn), VYVDURA results in the
reduction of circulating IgG. It is the first-and-only approved
FcRn blocker administered by subcutaneous injection for the
treatment of CIDP.
VYVDURA is the proprietary name in Japan for
subcutaneous efgartigimod alfa and recombinant human hyaluronidase
PH20. It is marketed under different proprietary names in other
regions.
About Chronic Inflammatory Demyelinating
Polyneuropathy
Chronic inflammatory demyelinating
polyneuropathy (CIDP) is a rare and serious autoimmune disease of
the peripheral nervous system. Although confirmation of disease
pathophysiology is still emerging, 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 get worse 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.
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 in the U.S.,
Japan, Israel, the EU, the UK, Canada and China. The Company is
evaluating efgartigimod in multiple serious autoimmune diseases and
advancing several earlier stage experimental medicines within its
therapeutic franchises. For more information,
visit www.argenx.com and follow us
on LinkedIn, Twitter, and Instagram.
Contacts
Media:
Ben PetokBpetok@argenx.com
Investors:
Alexandra Roy (US) aroy@argenx.com
Lynn Elton (EU) lelton@argenx.com
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