argenx Announces Approval of VYVDURA (efgartigimod alfa and hyaluronidase-qvfc) in Japan for Adults with Chronic Inflammatory Demyelinating Polyneuropathy
argenx announced that Japan's MHLW has approved VYVDURA for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The treatment is administered as a weekly 30-90 second subcutaneous injection that can be self-administered at home, making it the first FcRn blocker approved for CIDP treatment in Japan.
The approval is based on the ADHERE Study, which showed that 69% of VYVDURA-treated patients demonstrated clinical improvement, with a 61% reduction in relapse risk versus placebo. The study was the largest clinical trial for CIDP to date, with 99% of participants continuing in the extension phase.
This marks VYVDURA's third approved indication in Japan, following its earlier approval for generalized myasthenia gravis (gMG) in January 2024.
argenx ha annunciato che il MHLW giapponese ha approvato VYVDURA per adulti affetti da neuropatia periferica demielinizzante infiammatoria cronica (CIDP). Il trattamento viene somministrato come un'iniezione sottocutanea settimanale della durata di 30-90 secondi, che può essere auto-somministrata a casa, rendendolo il primo bloccatore FcRn approvato per il trattamento della CIDP in Giappone.
L'approvazione si basa sullo ADHERE Study, che ha dimostrato che il 69% dei pazienti trattati con VYVDURA ha mostrato un miglioramento clinico, con una riduzione del 61% del rischio di ricaduta rispetto al placebo. Lo studio è stato il più grande trial clinico per la CIDP fino ad oggi, con il 99% dei partecipanti che ha continuato nella fase di estensione.
Questo segna la terza indicazione approvata per VYVDURA in Giappone, dopo la sua precedente approvazione per il grave miastenia gravis (gMG) a gennaio 2024.
argenx anunció que el MHLW de Japón ha aprobado VYVDURA para adultos con polineuropatía desmielinizante inflamatoria crónica (CIDP). El tratamiento se administra como una inyección subcutánea semanal de 30-90 segundos que puede ser autoadministrada en casa, siendo el primer bloqueador de FcRn aprobado para el tratamiento de CIDP en Japón.
La aprobación se basa en el ADHERE Study, que mostró que el 69% de los pacientes tratados con VYVDURA presentaron mejoría clínica, con una reducción del 61% en el riesgo de recaída en comparación con el placebo. El estudio fue el más grande ensayo clínico para CIDP hasta la fecha, con el 99% de los participantes continuando en la fase de extensión.
Esto marca la tercera indicación aprobada para VYVDURA en Japón, después de su aprobación anterior para la miastenia gravis generalizada (gMG) en enero de 2024.
argenx는 일본 MHLW가 만성 염증성 탈수초성 다발신경병증(CIDP) 성인을 위한 VYVDURA를 승인했다고 발표했습니다. 이 치료는 30-90초 동안 주간 피하 주사로 제공되며 자가 주사가 가능하여 일본에서 CIDP 치료를 위해 승인된 첫 번째 FcRn 차단제가 됩니다.
승인은 ADHERE Study를 기반으로 하며, 이 연구에서는 VYVDURA 치료를 받은 환자의 69%가 임상적 개선을 보였고, 위약 대비 재발 위험이 61% 감소했습니다. 이 연구는 지금까지 CIDP를 위한 가장 큰 임상 시험이었고, 참가자의 99%가 연장 단계로 지속되었습니다.
이는 VYVDURA의 세 번째 승인 적응증으로, 2024년 1월에 일반화된 중증 근무력증(gMG)에 대해 이전에 승인된 바 있습니다.
argenx a annoncé que le MHLW du Japon a approuvé VYVDURA pour les adultes atteints de polynévrite démyélinisante inflammatoire chronique (CIDP). Le traitement est administré par une injection sous-cutanée hebdomadaire de 30 à 90 secondes pouvant être auto-administrée à domicile, ce qui en fait le premier bloqueur FcRn approuvé pour le traitement de la CIDP au Japon.
L'approbation est basée sur l'ADHERE Study, qui a montré que 69% des patients traités avec VYVDURA ont présenté une amélioration clinique, avec une réduction de 61% du risque de rechute par rapport au placebo. L'étude était le plus grand essai clinique pour la CIDP à ce jour, avec 99% des participants poursuivant dans la phase d'extension.
Cela marque la troisième indication approuvée pour VYVDURA au Japon, après son approbation antérieure pour la myasthénie grave généralisée (gMG) en janvier 2024.
argenx gab bekannt, dass das MHLW von Japan VYVDURA für Erwachsene mit chronischer entzündlicher demyelinisierender Polyneuropathie (CIDP) genehmigt hat. Die Behandlung wird als wöchentliche subkutane Injektion von 30-90 Sekunden verabreicht, die zu Hause selbst verabreicht werden kann, was es zum ersten FcRn-Blocker macht, der in Japan zur Behandlung von CIDP zugelassen wurde.
Die Genehmigung basiert auf der ADHERE-Studie, die zeigte, dass 69% der mit VYVDURA behandelten Patienten eine klinische Verbesserung zeigten, wobei das Risiko eines Rückfalls im Vergleich zur Placebo-Gruppe um 61% gesenkt wurde. Die Studie war die bisher größte klinische Prüfung für CIDP, wobei 99% der Teilnehmer in die Erweiterungsphase übergingen.
Dies markiert die dritte zugelassene Indikation für VYVDURA in Japan, nach der früheren Genehmigung für die generalisierte Myasthenia gravis (gMG) im Januar 2024.
- 69% of patients showed clinical improvement in the ADHERE Study
- 61% reduction in relapse risk compared to placebo
- First and only FcRn blocker approved for CIDP treatment in Japan
- Convenient 30-90 second at-home self-injection administration
- 99% trial participant retention in the ADHERE+ extension study
- None.
Insights
The MHLW approval of VYVDURA for CIDP in Japan marks a important milestone for argenx, expanding its commercial footprint in a key market. This represents the third approved indication in Japan for argenx's FcRn franchise, creating a significant first-mover advantage in treating multiple autoimmune conditions. The commercial opportunity is substantial, considering CIDP's chronic nature and the current lack of innovative treatments over the past three decades.
The 69% response rate and 61% reduction in relapse risk demonstrated in the ADHERE trial, coupled with the convenient subcutaneous administration, position VYVDURA favorably against existing treatments. The high retention rate in the extension study (99%) suggests strong patient satisfaction, which could drive rapid market adoption.
VYVDURA's approval addresses a critical unmet need in CIDP treatment. The current CIDP treatment landscape is , with 85% of patients requiring ongoing therapy and 88% experiencing residual impairment despite treatment. The 30-90 second self-administration feature significantly improves the treatment experience compared to traditional lengthy infusions.
The ADHERE trial's robust efficacy data, particularly the significant reduction in relapse risk, validates VYVDURA's clinical value. The consistent safety profile across multiple indications strengthens its position as a reliable treatment option. The Orphan Drug designation further underscores the therapeutic's importance in addressing this rare disease.
This approval strengthens argenx's competitive position in the
The first-mover advantage in three indications positions argenx to capture substantial market share before potential competitors enter. The strong efficacy data and convenient administration format could drive premium pricing and robust reimbursement coverage, potentially leading to significant revenue growth in the Japanese market.
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,
The MHLW approval is based on the ADHERE Study, the largest clinical trial to date studying CIDP. In the ADHERE study,
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 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
FAQ
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