Full Press Release Details
argenx Announces Approval of VYVGART (efgartigimod
alfa) in Japan for Adults with Primary Immune Thrombocytopenia
VYVGART now approved
in Japan for both generalized myasthenia gravis and primary immune thrombocytopenia (ITP)
Regulatory decision in Japan represents first
global approval for VYVGART in ITP
March 26, 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 VYVGART (efgartigimod alfa) for intravenous
(IV) use in adults with primary immune thrombocytopenia (ITP).
"argenx is on a mission to deliver transformative
medicines for people living with severe autoimmune disease," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We
have always believed VYVGART has the potential to treat many IgG-mediated autoimmune diseases, and now patients in Japan, who have been
waiting for a new treatment option, have one for ITP. My sincere thanks to all of those who contributed to today's milestone approval
in Japan - it takes true collaboration to bring real innovation for the millions of patients around the world living with rare autoimmune
disease. And our work has only just begun."
"ITP is a severe, debilitating autoimmune
disease, and ITP patients typically experience a significantly lower quality of life," said Luc Truyen, M.D.,
Ph.D., Chief Medical Officer of argenx. "Physicians
now treat ITP via a trial-and-error approach - or by cycling through current therapies. By reducing circulating autoantibodies,
VYVGART is uniquely designed to serve as a precision intervention that
targets the underlying disease biology of ITP. We are grateful to all of those who contributed to advancing our understanding of the disease
so that we could bring VYVGART to patients in Japan."
The approval of VYVGART is based on results from
the global Phase 3 ADVANCE-IV trial, which were published in the September 2023 issue of The Lancet. ADVANCE successfully
met its primary endpoint, demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet
count response compared to placebo. VYVGART demonstrated rapid onset of effect in chronic and persistent ITP patients, as well as a 51%
response rate on the International Working Group (IWG) score, which is a measure developed by the world's leading experts on ITP
and highly relevant to clinical care. Primary endpoint responders were observed across patient types regardless of age, disease severity,
time since diagnosis, prior ITP treatment or background medication. VYVGART was well-tolerated in this 24-week study and the observed
safety and tolerability profile was consistent with previous clinical trials.
Phase 3 ADVANCE Trial
The ADVANCE trial enrolled 131 adult patients
with chronic and persistent ITP. Patients were heavily pretreated and 67% of patients had received three or more prior ITP therapies,
including 59% who had prior thrombopoietin receptor agonist (TPO-RAs) experience, 34% with prior rituximab experience and 37% with a history
of splenectomy. Patients were insufficiently controlled at baseline with mean platelet counts of 17x109/L across all patients.
Of patients who completed the full ADVANCE study, 94% (63/67) of VYVGART-treated patients and 97% (38/39) of placebo patients continued
to the ADVANCE+ open-label extension study.
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.
What is VYVGART (efgartigimod
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).
IMPORTANT SAFETY INFORMATION
Do not use VYVGART if you have a serious allergy
to efgartigimod alfa or any of the other ingredients in VYVGART. VYVGART can cause serious allergic reactions and a decrease in blood
pressure leading to fainting.
VYVGART may cause serious side effects, including:
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 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
Before taking VYVGART, tell your doctor if
What are the common side effects of VYVGART?
The most common side effects of VYVGART are respiratory
tract infection, headache, and urinary tract infection. These are not all 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.
Please see the full Prescribing Information
for VYVGART and talk to your doctor.
About Immune Thrombocytopenia
Immune thrombocytopenia (ITP) is an autoimmune
disorder where immunoglobulin G (IgG) autoantibodies destroy platelets and reduce platelet production, which can lead to an increased
risk of excessive bleeding and bruising. In severe cases, frequent bleeding events can cause anemia or even brain hemorrhage in rare cases.
ITP is also associated with debilitating fatigue and significant impacts on mental health, including anxiety, fear and depression. Many
ITP patients are inadequately controlled on current therapies so there remains a significant unmet need for additional treatment options.
About VYVGART (efgartigimod
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 is being studied in adults with primary
immune thrombocytopenia (ITP) and other IgG autoantibody-mediated diseases. VYVGART has been approved for adults with ITP only in Japan
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
Forward-Looking Statements
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 "aims," "believe,"
"committed" or "potential" and include statements argenx makes concerning its mission to deliver transformative
medicines for people living with severe autoimmune disease; its belief that VYVGART has the potential to treat many IgG-mediated autoimmune
diseases such as ITP; the potential impact of VYVGART for ITP patients; 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 development of novel drug therapies, preclinical and clinical trial and product development activities and regulatory approval requirements,
the acceptance of our products and product candidates by our patients as safe, effective and cost-effective, and the impact of governmental
laws and regulations on our business. 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.