argenx Announces Approval of VYVGART (efgartigimod alfa) in Japan for Adults with Primary Immune Thrombocytopenia
- Regulatory approval in Japan for VYVGART to treat primary immune thrombocytopenia (ITP)
- Phase 3 ADVANCE-IV trial results led to approval based on efficacy in chronic ITP patients
- VYVGART showed a rapid onset of effect and a well-tolerated safety profile
- Global approval in Japan signifies a significant milestone for argenx SE and VYVGART
- Precision intervention targeting the underlying disease biology of ITP
- None.
Insights
The approval of VYVGART in Japan for the treatment of primary immune thrombocytopenia (ITP) marks a significant development in the field of autoimmune disease management. ITP is a chronic condition characterized by a dangerously low platelet count, leading to excessive bruising, bleeding and other serious health issues. The traditional approach to treating ITP often involves a degree of guesswork, with physicians cycling through various therapies to find an effective treatment. The introduction of VYVGART, which operates by reducing circulating autoantibodies, represents a more targeted strategy that aligns with the precision medicine paradigm.
From a research perspective, the positive outcome of the Phase 3 ADVANCE-IV trial, which showed a 51% response rate on the IWG score, is particularly notable. This response rate is significant as it suggests a substantial proportion of patients treated with VYVGART experienced improved platelet counts, which is a direct measure of therapeutic efficacy. Additionally, the fact that the drug was well-tolerated across a diverse patient population, including those with extensive prior treatments, positions VYVGART as a potentially valuable addition to the existing ITP treatment landscape.
The approval of VYVGART for ITP in Japan is not only a medical milestone but also an event with economic implications. The Japanese healthcare market is known for its rigorous regulatory standards and an approval here often paves the way for subsequent approvals in other countries, potentially leading to a broadened market presence for argenx SE. For stakeholders, this expansion in indications could mean a significant increase in the patient pool eligible for treatment with VYVGART, thus potentially increasing the drug's revenue stream.
In terms of cost-effectiveness, precision therapies like VYVGART can lead to better resource allocation in healthcare by reducing the need for trial-and-error treatment approaches, which are often time-consuming and expensive. However, the high cost of novel biologics is a concern that payers and health systems must consider. The long-term economic impact will depend on the drug's pricing strategy, reimbursement policies and the overall reduction in healthcare utilization if the therapy proves to reduce the frequency and severity of ITP-related complications.
The pharmaceutical landscape for autoimmune diseases is highly competitive, with numerous companies vying for market share. The approval of VYVGART for ITP in Japan represents a strategic advantage for argenx SE, as it is the first global approval for the drug in this indication. This first-mover advantage could be instrumental in establishing the company as a leader in the treatment of IgG-mediated diseases.
Investors should monitor the uptake of VYVGART in Japan closely, as it may serve as an indicator of the drug's potential performance in other markets. Furthermore, the successful transition of patients from the ADVANCE trial to the ADVANCE+ open-label extension study suggests strong patient and clinician interest in VYVGART, which could translate into robust market adoption. Analysts will be looking at prescription trends, market penetration rates and competitive responses to assess the impact on argenx SE's financial performance and stock valuation.
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
Phase 3 ADVANCE Trial
The ADVANCE trial enrolled 131 adult patients with chronic and persistent ITP. Patients were heavily pretreated and
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 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).
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:
- Infection. VYVGART may increase the risk of infection. The most common infections 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 can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with VYVGART.
- Infusion-Related Reactions. VYVGART can cause infusion-related reactions. The most frequent symptoms and signs reported with VYVGART 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 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, 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?
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 alfa-fcab)
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 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 at this time.
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 Petok
Bpetok@argenx.com
Investors:
Alexandra Roy (US)
aroy@argenx.com
Lynn Elton (EU)
lelton@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 “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.
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