Full Press Release Details
Biologics License Application to U.S. Food and Drug Administration for Subcutaneous Efgartigimod for Treatment of Generalized Myasthenia
Netherlands - September 21, 2022 - argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to
improving the lives of people suffering from severe autoimmune diseases, today announced the submission of a Biologics License Application
(BLA) to the U.S. Food and Drug Administration (FDA) for SC efgartigimod (1000mg efgartigimod-PH20) for the treatment of generalized
myasthenia gravis (gMG) in adult patients.
is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE drug delivery technology. ENHANZE
facilitates the subcutaneous injection delivery of biologics that are typically administered via intravenous (IV) infusion.
for the gMG program is to deliver the broadest treatment offering for people living with this debilitating, and often overlooked disease.
Every individual experiences gMG differently, which is why we're excited about the possibility of introducing multiple ways to
meet the needs of patients, including with route of administration and dosing schedule," said Tim Van Hauwermeiren, Chief Executive
Officer of argenx. "The submission of this BLA is the latest milestone in honoring our commitment to the gMG patient community.
We look forward to working closely with the agency through the BLA review process and to potentially bringing forth another first-in-class
option for gMG patients."
package includes data from the Phase 3 ADAPT-SC study evaluating the noninferiority of the pharmacodynamic (PD) effect of SC efgartigimod
as compared with intravenously administered VYVGART in adult patients with gMG. The majority of enrolled patients were positive for acetylcholine
receptor (AChR) antibodies, but the trial also included patients where AChR antibodies were not detected.
primary endpoint (p< 0.0001) of total IgG reduction from baseline at day 29 demonstrating noninferiority of SC efgartigimod to VYVGART.
Patients treated with SC efgartigimod achieved mean total IgG reduction of 66.4% from baseline at day 29, compared to 62.2% reduction
with VYVGART. Results were consistent across the overall population, including those with AChR antibodies and patients where AChR antibodies
were not detected. Further, 69.1% of patients treated with SC efgartigimod were responders on the Myasthenia Gravis Activities of Daily
Living (MG-ADL) score. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive
weeks. 65.5% of patients treated with SC efgartigimod were responders on the Quantitative Myasthenia Gravis (QMG) score. Responders are
defined as having at least a three-point improvement on the QMG score for at least four consecutive weeks. Minimal symptom expression
(MSE), a measure of symptom-free status, was achieved in 37% of SC efgartigimod-treated patients after one treatment cycle. Onset of
effect was also consistent with the Phase 3 ADAPT study.
for SC efgartigimod was consistent with the ADAPT study. It was generally well-tolerated; the most frequent adverse event being injection
site reactions (ISRs), commonly observed with biologics administered subcutaneously. All ISRs were mild to moderate and resolved over
time. After completing ADAPT-SC, 95% of participants entered ADAPT-SC+, a three-year open-label extension study evaluating the long-term
safety and tolerability of SC efgartigimod.
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 (1000mg efgartigimod-PH20) as compared with VYVGART (10mg/kg) in patients with gMG. The pharmacodynamic effect as
measured by percent change from baseline in total IgG levels at day 29, one week after the last dose of IV or SC efgartigimod, served
as the primary endpoint in the ADAPT-SC trial. The correlation between total IgG reduction and clinical benefit in gMG was demonstrated
in a Phase 2 trial and the Phase 3 ADAPT trial, which served as the basis for approval of VYVGART in the U.S., Japan and Europe. Safety,
clinical efficacy, immunogenicity and pharmacokinetics (PK) were also assessed.
adult patients with gMG in North America, Europe and Japan enrolled in the ADAPT-SC trial and were treated. Inclusion criteria of the
trial were the same as the Phase 3 ADAPT trial of VYVGART; enrolled patients had a confirmed gMG diagnosis and an MG-ADL total score
of at least 5 with greater than 50% of the total score attributed to non-ocular symptoms, at screening and baseline. Patients were on
a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal
immunosuppressive drugs, and were required to remain on that stable dose throughout the primary trial. Patients were eligible to enroll
in ADAPT-SC regardless of antibody status, including patients with AChR antibodies (AChR-Ab+) and patients where AChR antibodies were
Patients were randomized
in a 1:1 ratio to receive SC efgartigimod or IV efgartigimod for one treatment cycle consisting of four doses at weekly intervals. The
total study duration was approximately 12 weeks, including seven weeks of follow-up after the treatment cycle.
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 for VYVGART (efgartigimod alfa-fcab) intravenous (IV)
formulation (U.S. prescribing information)
(efgartigimod alfa-fcab)?
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).
most important information I should know about VYVGART?
serious side effects, including:
Before taking VYVGART,
tell your health care provider about all of your medical conditions, including if you:
care provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
common side effects of VYVGART?
The most common side effects of VYVGART
are respiratory tract infection, headache, and urinary tract infection.
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 1-800-FDA-1088.
full Prescribing Information for VYVGART and talk to your doctor.
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 intravenous and subcutaneous
(SC) formulation. SC efgartigimod is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE
drug delivery technology.
(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 and Europe 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
Generalized Myasthenia Gravis
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 population1.
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 and the EU. 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
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 submission of the Biologics License Application to the U.S. Food and Drug Administration
for Subcutaneous (SC) Efgartigimod for Treatment of Generalized Myasthenia Gravis and the long-term safety and tolerability of SC Efgartigimod.
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 publicly update or revise the information in this press
release, including any forward-looking statements, except as may be required by law.