Full Press Release Details
argenx Highlights Data Evaluating VYVGART in
Neuromuscular Autoimmune Disease at AANEM and MGFA Scientific Sessions
Long-term clinical trial and real-world data
illustrate VYVGART drives consistent, repeatable and clinically meaningful responses, including minimal symptom
expression (MSE) in generalized myasthenia gravis (gMG)
Patients treated with VYVGART experienced consistent
improvements on key quality of life measures based on long-term gMG extension data
Data across multiple indications and dosing
schedules confirm favorable safety profile and no increase in treatment-emergent adverse event rates with longer exposure
Amsterdam, the Netherlands - Nov. 1,
2023 - argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering
from severe autoimmune diseases, today announced 20 presentations of clinical trial and real-world data from studies of VYVGART
and VYVGART Hytrulo (VYVGART) in neuromuscular autoimmune disease. The data presentations will be featured at the
American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Annual Meeting and the Myasthenia Gravis Foundation of America
(MGFA) Scientific Session, taking place in Phoenix, AZ from November 1-4, 2023.
"As leaders in the field of FcRn inhibition, we continue to generate
deep and broad data from our clinical trials, including long-term extension and real-world evidence studies," said Luc Truyen, M.D.,
Ph.D., Chief Medical Officer at argenx. "With VYVGART for gMG, we continue to show favorable safety and consistent, repeatable clinically
meaningful responses, including the ability of patients to achieve MSE, across three years of treatment. The ADHERE study, the largest
in the history of CIDP, further demonstrates our commitment to the neuromuscular community. These data characterize the broad impact our
first-in-class FcRn blocker can have on the lives of people living with CIDP, and our work to establish its safety and efficacy in the
treatment of CIDP represents an exciting step forward for this community."
Highlights from Data Presented at AANEM and
Additional data are being presented from case
studies of gMG patients with LRP4 antibodies, a meta-analysis of quality-of-life outcomes of VYVGART and other gMG treatments, and argenx-sponsored
health economic outcomes research studies demonstrating gMG patients facing social-determinants of health challenges experience health
inequities related to increased utilization of acute care facilities, and delayed diagnosis and access to treatment.
AANEM Poster Presentations (November 1-4, 2023)
| # | Title | Lead Author | Presentation |
| 143 | Efficacy, Safety, And Tolerability Of Efgartigimod In Patients With Chronic Inflammatory Demyelinating Polyneuropathy: Results From The Adhere Trial | Richard Lewis, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session II Nov. 3 9:30 AM - 10:00 AM MST |
| 151 | Long-Term Safety, Tolerability, and Efficacy of Subcutaneous Efgartigimod PH20 in Participants With Generalized Myasthenia Gravis: Interim Analysis of Anti-Acetylcholine Receptor Autoantibody Seronegative Participants in the ADAPT-SC+ Study | James F. Howard Jr., MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
| 270 | Dose Selection and Clinical Development Of Efgartigimod PH20 Subcutaneous In Patients With Generalized Myasthenia Gravis | Tuan Vu, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session II Nov. 3 9:30 AM - 10:00 AM MST |
| 222 | Long-Term Safety, Tolerability, and Efficacy of Subcutaneous Efgartigimod PH20 in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT-SC+ Study | Yuebing Li, MD, PhD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
| 152 | Long-Term Safety, Tolerability, and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Concluding Analyses From the ADAPT+ Study | James F. Howard Jr., MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
| 240 | A Case of Treatment With Efgartigimod in a Patient With Generalized Myasthenia Gravis and LRP4 Antibodies | Eduardo De Sousa, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session II Nov. 3 9:30 AM - 10:00 AM MST |
| 220 | Overview Of The Safety Profile From Efgartigimod Clinical Trials In Participants With Diverse Immunoglobulin G-Mediated Autoimmune Diseases | Kelly Gwathmey, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
| 139 | Treatment-Related Inequities in Patients With Generalized Myasthenia Gravis Facing Social Determinants of Health Challenges: A Survey of Neurologists in the United States | A. Gordon Smith, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
| 138 | Diagnosis Inequities In Patients With Generalized Myasthenia Gravis Facing Social Determinants Of Health Challenges: A Survey Of Neurologists In The United States | A. Gordon Smith, MD | Poster Session I Nov. 2 6:00 PM - 6:30 PM MST Poster Session III Nov. 3 3:00 PM - 3:30 PM MST |
MGFA Oral and Poster Presentations (November 1, 2023)
| Title | Lead Author | Presentation |
| Achievement of Minimal Symptom Expression in Acetylcholine-Receptor Antibody-Positive Participants with Generalized Myasthenia Gravis and Effect on Disease-Specific Measures in ADAPT/ADAPT+ Studies | James F. Howard, MD | Oral Presentation 10:07 AM - 10:14 AM MST |
| Subcutaneous Efgartigimod PH20 Treatment in Participants With Generalized Myasthenia Gravis in ADAPT-SC+: Interim Analyses on Quality of Life, Efficacy, Tolerability, and Long-Term Safety | Tuan Vu, MD | Oral Presentation 10:42 AM - 10:49 AM MST |
| Racial Disparities in Acute Care Utilization Outcomes Among Those with Myasthenia Gravis | Pushpa Narayanaswami, MD | Oral Presentation 9:07 AM - 9:14 AM MST |
| Humoral Immune Response to Polyvalent Pneumococcal Vaccine in Healthy Participants Receiving Efgartigimod | Antoine Azar, MD | Poster Session 9:35 AM - 10:05 AM MST |
| Network Meta Analysis of Treatment Options in Generalized Myasthenia Gravis: Impact on Health-Related Quality of Life | Gil Wolfe, MD | Poster Session 9:35 AM - 10:05 AM MST |
| Overview of the Safety Profile From Efgartigimod Clinical Trials in Participants with Diverse IgG-mediated Autoimmune Diseases | Kelly Gwathmey, MD | Poster Session 9:35 AM - 10:05 AM MST |
| Treatment-Related Inequities in Patients With Generalized Myasthenia Gravis Facing Social Determinants of Health Challenges: A Survey of Neurologists in the United States | A. Gordon Smith, MD | Poster Session 9:35 AM - 10:05 AM MST |
| Diagnostic Inequities in Patients With Generalized Myasthenia Gravis Facing Social Determinants of Health Challenges: A Survey of Neurologists in the United States | A. Gordon Smith, MD | Poster Session 9:35 AM - 10:05 AM MST |
| Real-World Burden Associated With Social Determinants of Health Challenges For Individuals Living with Generalized Myasthenia Gravis in the United States | Tom Hughes | Poster Session 9:35 AM - 10:05 AM MST |
| Real-World Outcomes of Patients living with Generalized Myasthenia Gravis Initiating Efgartigimod treatment in the United States | Cynthia Qi | Poster Session 9:35 AM - 10:05 AM MST |
| Association between patient support program participation and access to efgartigimod treatment for generalized myasthenia gravis | Glenn Phillips | Poster Session 9:35 AM - 10:05 AM MST |
More information on the programs are available at AANEM
See Important Safety Information below and
full Prescribing Information for VYVGART Hytrulo for additional information
Important Safety Information
Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)?
VYVGART Hytrulo 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).
IMPORTANT SAFETY INFORMATION
VYVGART and VYVGART HYTRULO
may increase the risk of infection. The most common infections observed in Study 1 were urinary tract infection (10% of efgartigimod alfa-fcab-treated
patients vs 5% of placebo-treated patients) and respiratory tract infection (33% of efgartigimod alfa-fcab-treated patients vs 29% of
placebo-treated patients). Patients on efgartigimod alfa-fcab vs placebo had below normal levels for white blood cell counts (12% vs 5%,
respectively), lymphocyte counts (28% vs 19%, respectively), and neutrophil counts (13% vs 6%, respectively). The majority of infections
and hematologic abnormalities were mild to moderate in severity. Delay the administration of VYVGART or VYVGART HYTRULO in patients with
an active infection until the infection has resolved; monitor for clinical signs and symptoms of infections. If serious infection occurs,
administer appropriate treatment and consider withholding treatment with VYVGART or VYVGART HYTRULO until the infection has resolved.
Immunization with vaccines
during treatment with VYVGART or VYVGART HYTRULO has not been studied; the safety with live or live-attenuated vaccines and the response
to immunization with any vaccine are unknown. Because VYVGART and VYVGART HYTRULO cause a reduction in immunoglobulin G (IgG) levels,
vaccination with live-attenuated or live vaccines is not recommended during treatment with VYVGART or VYVGART HYTRULO. Evaluate the need
to administer age-appropriate vaccines according to immunization guidelines before initiation of a new treatment cycle with VYVGART or
Hypersensitivity Reactions
Hypersensitivity reactions,
including rash, angioedema, and dyspnea were observed in patients treated with VYVGART or VYVGART HYTRULO. Urticaria was also observed
in patients treated with VYVGART HYTRULO. In clinical trials, hypersensitivity reactions were mild or moderate, occurred within 1 hour
to 3 weeks of administration, and did not lead to treatment discontinuation. Monitor patients during and for one hour after VYVGART administration,
or for at least 30 minutes after VYVGART HYTRULO administration, for clinical signs and symptoms of hypersensitivity reactions. If a hypersensitivity
reaction occurs during VYVGART or VYVGART HYTRULO administration, discontinue use and institute appropriate supportive measures if needed.
In Study 1, the most
common ( 10%) adverse reactions in efgartigimod alfa-fcab-treated patients were respiratory tract infection, headache, and urinary
tract infection. In Study 2, the most common ( 10%) adverse reactions in VYVGART HYTRULO-treated patients were injection site reactions
and headache. Injection site reactions occurred in 38% of VYVGART HYTRULO-treated patients, including injection site rash, erythema, pruritus,
bruising, pain, and urticaria. In Study 2 and its open-label extension, all injection site reactions were mild to moderate in severity
and did not lead to treatment discontinuation. The majority occurred within 24 hours after administration and resolved spontaneously.
Most injection site reactions occurred during the first treatment cycle, and the incidence decreased with each subsequent cycle.
USE IN SPECIFIC POPULATIONS
As VYVGART and VYVGART
HYTRULO are expected to reduce maternal IgG antibody levels, reduction in passive protection to the newborn is anticipated. Risks and
benefits should be considered prior to administering live or live attenuated vaccines to infants exposed to VYVGART or VYVGART HYTRULO
There is no information
regarding the presence of efgartigimod alfa-fcab from administration of VYVGART, or efgartigimod alfa or hyaluronidase from administration
of VYVGART HYTRULO, in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health
benefits of breastfeeding should be considered along with the mother's clinical need for VYVGART or VYVGART HYTRULO, and any potential
adverse effects on the breastfed infant from VYVGART or VYVGART HYTRULO or from the underlying maternal condition.
VYVGART (efgartigimod
alfa-fcab) for intravenous infusion and VYVGART HYTRULO (efgartigimod alfa and hyaluronidase-qvfc) for subcutaneous
injection are each indicated for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor
(AChR) antibody positive.
Please see the full Prescribing
Information for VYVGART and the full Prescribing Information for VYVGART HYTRULO.
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,1 where muscles throughout
the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population.1
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.
VYVGART is a human IgG1
antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. It is the
first approved FcRn blocker in the United States, EU and China 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 therapies (ISTs).
VYVGART Hytrulo is a