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 INFORMATION
Do 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 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
VYVDURA現在在日本獲得批准用於家庭自我注射，適用於廣泛型重症肌無力和CIDP患者
argenx的VYVGARt和VYVDURA產品組合在日本獲得三項適應症的批准——這是全球首個在三項適應症上獲得的國家
2024年12月27日，上午7:00 CET
荷蘭阿姆斯特丹——argenx SE（Euronext & 納斯達克：ARGX），一家全球免疫學公司，致力於改善重症自身免疫疾病患者的生活，今天宣佈日本衛生勞動福利部（MHLW）已批准VYVDURA用於治療慢性炎症性脫髓鞘性多神經病（CIDP）成年人。VYVDURA獲得批准用於CIDP，作爲每週一次的30至90秒皮下注射，可以在家自我施用，是第一款也是唯一一款獲得批准用於CIDP治療的 neonatal Fc 受體（FcRn）阻斷劑。
"CIDP是一種罕見且嚴重的疾病，過去30年在治療方面幾乎沒有創新，" argenx的首席醫療官Luc Truyen萬.D.博士表示。"通過VYVDURA，日本的CIDP患者現在可以接受一種新療法，這種療法的作用機制集中，提供方便的30到90秒的居家自我注射選項，並具有既往證實的療效和安全性，如ADHERE試驗和現實世界證據所示。通過將這種變革性治療的覆蓋範圍延伸到成千上萬的患者，argenx繼續將我們的首個FcRn抑制劑efgartigimod帶給在日本及全球範圍內遭受嚴重自身免疫疾病的患者。"
CIDP是一種進行性免疫介導的罕見且嚴重的周圍神經系統肌肉疾病。患者會經歷一系列導致活動和感覺問題的症狀，包括從坐姿站起時的困難、疼痛和疲勞，以及頻繁絆倒或摔倒。許多患者最終只能依賴輪椅，隨着疾病的發展，無法工作。目前，85%的患者需要持續治療，近88%的接受治療的患者經歷殘餘損傷和殘疾。
MHLW的批准基於ADHERE研究，該研究是迄今爲止研究CIDP的最大臨牀試驗。在ADHERE研究中，無論以往治療如何，69%（221/322）的VYVDURA治療患者表現出臨牀改善的證據，包括活動、功能和力量的改善。ADHERE滿足其主要終點（p<0.0001），表明相較於安慰劑，復發風險減少61%（HR: 0.39 95% CI: 0.25; 0.61）。99%的試驗參與者選擇參與ADHERE+開放標籤延續研究。安全性結果與之前臨牀研究和現實世界使用中已知的VYVDURA安全性特徵普遍一致。
VYVDURA於2024年1月獲得MHLW批准進行生產和營銷，並於2024年4月上市用於治療廣泛性重症肌無力（gMG）。在2024年3月，VYVDURA被MHLW指定爲治療CIDP的孤兒藥。
請查看以下FDA批准的重要安全信息及VYVDURA的完整處方信息，VYVDURA在美國以VYVGARt Hytrulo的名稱銷售，以獲取更多信息。
什麼是VYVGARt HYTRULO（efgartigimod ALFA和透明質酸酶-qvfc）？
VYVGARt HYTRULO是一種處方藥，用於治療患有慢性炎症性脫髓鞘多發性神經病（CIDP）的成年患者。
重要安全信息
如果您對efgartigimod ALFA、透明質酸酶或VYVGARt HYTRULO中的任何其他成分有嚴重過敏反應，請不要使用VYVGARt HYTRULO。VYVGARt HYTRULO可能會引起嚴重的過敏反應和血壓降低，導致暈厥。
VYVGARt HYTRULO可能會引起嚴重的副作用，包括：
- 感染。VYVGARt HYTRULO可能會增加感染的風險。接受efgartigimod ALFA-fcab治療的患者中，最常見的感染是尿路感染和呼吸道感染。感染的跡象或症狀可能包括髮熱、寒戰、頻繁和/或疼痛性排尿、咳嗽、鼻道/竇道疼痛和阻塞、喘息、呼吸急促、疲勞、喉嚨痛、痰多、鼻涕、背痛和/或胸痛。
- 過敏反應（超敏反應）。VYVGARt HYTRULO可以引起過敏反應，如皮疹、皮下腫脹和呼吸急促。在接受VYVGARt HYTRULO治療的患者中也觀察到了蕁麻疹。嚴重的過敏反應，如呼吸困難和血壓下降導致的暈厥，已在efgartigimod ALFA-fcab的報告中出現。
- 輸注相關反應。VYVGARt HYTRULO可以引起輸注相關反應。報告efgartigimod ALFA-fcab時，最常見的症狀和體徵是高血壓、寒戰、發抖，以及胸部、腹部和背部疼痛。
如果您有感染、過敏反應或輸注相關反應的跡象或症狀，請告訴醫生。這些可能在您接受VYVGARt HYTRULO治療期間或之後發生。您的醫生可能需要暫停或停止您的治療。如果您有嚴重過敏反應的跡象或症狀，請立即聯繫醫生。
在服用VYVGARt HYTRULO之前，請告知您的醫生如果您：
- 服用任何藥物，包括處方藥和非處方藥、補充劑或草藥藥物，
- 曾接受或計劃接受生物-疫苗（免疫接種），或者
- 有任何過敏或醫療狀況，包括您是否懷孕或計劃懷孕，或正在哺乳。
VYVGARt HYTRULO的常見副作用是什麼？
在efgartigimod-ALFA-fcab治療的患者中，最常見的副作用是呼吸道感染、頭痛和尿路感染。VYVGARt HYTRULO的其他常見副作用包括注射部位反應，如皮疹、皮膚紅腫、瘙癢感、淤傷、疼痛和蕁麻疹。
這些不是VYVGARt HYTRULO可能的所有副作用。請諮詢醫生獲取關於副作用的醫療建議。您可以通過撥打1-800-FDA-1088向美國食品和藥品管理局報告副作用。
請查看VYVGARt HYTRULO的完整處方信息並與醫生交談。
關於ADHERE試驗設計
ADHERE試驗是一項多中心、隨機、雙盲、安慰劑對照試驗，評估VYVDURA（efgartigimod ALFA和透明質酸酶-qvfc）用於治療慢性炎症性脫髓鞘多發性神經病（CIDP）。ADHERE招募了322名未接受治療（在過去六個月內未接受活躍治療或新確診）或正在接受免疫球蛋白治療或皮質類固醇治療的CIDP成年患者。該試驗由開放標籤的A階段和隨機的、安慰劑對照的B階段組成。爲了有資格參加該試驗，CIDP的診斷必須由獨立專家小組確認。患者進入一個預備階段，在該階段停止任何正在進行的CIDP治療，並且爲了有資格進入A階段，必須表現出活躍的疾病，即在至少一個CIDP臨牀評估工具（包括INCA、I-RODS或平均握力）上有臨牀有意義的惡化。未治療的患者可在近期惡化的證明下跳過預備期。爲了進入B階段，患者需要展示VYVDURA的臨牀改善證據（ECI）。ECI是通過改善INCA評分或在預備期中如果這些量表表現出惡化，改善I-RODS或平均握力獲得的。在B階段，患者被隨機分配至VYVDURA或安慰劑，持續時間最長可達48周。主要終點是在B階段總計達到88次復發或事件後測量，並基於首次調整的INCA惡化（即復發）所需的時間的風險比。在B階段之後，所有患者都有選項滾動進入一個開放標籤的擴展研究，以接受VYVDURA。
關於VYVDURA
VYVDURA是一種皮下給藥組合，由efgartigimod ALFA（一個人類IGG1抗體片段，以VYVGARt的名義以靜脈使用）和重組人透明質酸酶PH20（rHuPH20）組成，Halozyme的ENHANZE藥物遞送技術以促進生物製品的皮下注射給藥。VYVDURA通過結合新生兒Fc受體（FcRn）來減少循環中的IGG。這是第一個也是唯一一個以皮下注射方式給藥的FcRn攔截劑，用於治療CIDP。
VYVDURA是日本用於皮下efgartigimod ALFA和重組人透明質酸酶PH20的專有名稱。在其他地區以不同的專有名稱進行銷售。
關於慢性炎症性脫髓鞘多發性神經病
慢性炎症性脫髓鞘多發性神經病（CIDP）是一種罕見且嚴重的周圍神經系統自身免疫疾病。儘管該疾病的病理生理確認還在不斷髮展，但越來越多的證據表明，IGG抗體在周圍神經的損傷中起着關鍵作用。CIDP患者會感到疲勞、肌肉無力以及手臂和腿部的感覺喪失，這些症狀可能會隨着時間的推移而加重，或者時好時壞。這些症狀會顯著影響一個人在日常生活中的功能。沒有治療的話，三分之一的CIDP患者將需要使用輪椅。
關於argenx
argenx是一家全球免疫學公司，致力於改善遭受嚴重自身免疫疾病患者的生活。通過其免疫學創新計劃（IIP）與領先的學術研究者合作，argenx旨在將免疫學的突破轉化爲一流的抗體基礎新藥組合。argenx開發並在美國、日本、以色列、歐盟、英國、加拿大和中國商業化了首個獲批的胎兒Fc受體（FcRn）阻滯劑。公司正在評估efgartigimod在多種嚴重自身免疫疾病中的應用，並加快多個早期實驗藥物的開發。欲了解更多信息，請訪問並關注我們的LinkedIn、Twitter和Instagram。
聯繫人
媒體：
本·佩托克
Bpetok@argenx.com
投資者：
亞歷桑德拉·羅伊（美國）
aroy@argenx.com
林恩·埃爾頓（歐洲）
lelton@argenx.com
