argenx Announces Positive CHMP Opinion for VYVGART (efgartigimod alfa) Subcutaneous Injection for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
argenx has received a positive CHMP opinion recommending European Commission approval of VYVGART (efgartigimod alfa) for subcutaneous injection in treating chronic inflammatory demyelinating polyneuropathy (CIDP). The treatment represents the first novel mechanism of action for CIDP in over 30 years.
The recommendation is based on the ADHERE clinical trial, which demonstrated that 66.5% of patients showed clinical improvement. The study met its primary endpoint with a 61% reduction in relapse risk versus placebo. The treatment can be administered by patients, caregivers, or healthcare professionals, with an initial weekly dosing that may be adjusted to every other week based on clinical evaluation.
VYVGART showed consistent safety results with previous clinical studies, and 99% of trial participants continued into the open-label extension. The European Commission's final decision on marketing authorization is expected within approximately two months.
argenx ha ricevuto un parere positivo dal CHMP che raccomanda l'approvazione da parte della Commissione Europea di VYVGART (efgartigimod alfa) per iniezione sottocutanea nel trattamento della polineuropatia infiammatoria demielinizzante cronica (CIDP). Questo trattamento rappresenta il primo nuovo meccanismo d'azione per la CIDP in oltre 30 anni.
La raccomandazione si basa sul trial clinico ADHERE, che ha dimostrato un miglioramento clinico nel 66,5% dei pazienti. Lo studio ha raggiunto il suo endpoint primario con una riduzione del 61% del rischio di recidiva rispetto al placebo. Il trattamento può essere somministrato dai pazienti stessi, dai caregiver o dai professionisti sanitari, con una dose iniziale settimanale che può essere modificata a ogni due settimane in base alla valutazione clinica.
VYVGART ha mostrato risultati di sicurezza coerenti con studi clinici precedenti e il 99% dei partecipanti al trial ha proseguito nell'estensione in aperto. La decisione finale della Commissione Europea sull'autorizzazione alla commercializzazione è prevista entro circa due mesi.
argenx ha recibido una opinión positiva del CHMP que recomienda la aprobación por parte de la Comisión Europea de VYVGART (efgartigimod alfa) para inyección subcutánea en el tratamiento de la polineuropatía inflamatoria desmielinizante crónica (CIDP). Este tratamiento representa el primer mecanismo de acción novedoso para la CIDP en más de 30 años.
La recomendación se basa en el ensayo clínico ADHERE, que demostró que el 66,5% de los pacientes mostraron mejoría clínica. El estudio alcanzó su objetivo primario con una reducción del 61% en el riesgo de recaída frente al placebo. El tratamiento puede ser administrado por los pacientes, cuidadores o profesionales de la salud, con una dosificación inicial semanal que puede ajustarse a cada dos semanas según la evaluación clínica.
VYVGART mostró resultados de seguridad consistentes con estudios clínicos previos, y el 99% de los participantes continuaron en la extensión de etiqueta abierta. La decisión final de la Comisión Europea sobre la autorización de comercialización se espera en aproximadamente dos meses.
argenx가 만성 염증성 탈수초성 다발신경병증(CIDP) 치료를 위한 피하 주사제 VYVGART(에프가르티기모드 알파)의 유럽위원회 승인을 권고하는 CHMP의 긍정적인 의견을 받았습니다. 이 치료법은 CIDP에 대해 30년 만에 처음으로 새로운 작용 기전을 제시합니다.
이 권고는 66.5%의 환자가 임상적 개선을 보인 ADHERE 임상시험에 근거합니다. 연구는 위약 대비 재발 위험을 61% 감소시키는 1차 평가변수를 달성했습니다. 치료는 환자, 보호자 또는 의료진이 투여할 수 있으며, 초기 주간 투여 후 임상 평가에 따라 격주 투여로 조정될 수 있습니다.
VYVGART는 이전 임상 연구들과 일관된 안전성 결과를 보였으며, 시험 참가자의 99%가 공개 라벨 연장 연구에 참여했습니다. 유럽위원회의 최종 시판 허가 결정은 약 2개월 내에 예상됩니다.
argenx a reçu un avis positif du CHMP recommandant l'approbation par la Commission européenne de VYVGART (efgartigimod alfa) pour injection sous-cutanée dans le traitement de la polyradiculonévrite inflammatoire démyélinisante chronique (CIDP). Ce traitement représente le premier nouveau mécanisme d'action pour la CIDP depuis plus de 30 ans.
La recommandation s'appuie sur l'essai clinique ADHERE, qui a démontré une amélioration clinique chez 66,5 % des patients. L'étude a atteint son critère principal avec une réduction de 61 % du risque de rechute par rapport au placebo. Le traitement peut être administré par les patients, les aidants ou les professionnels de santé, avec une posologie hebdomadaire initiale pouvant être ajustée à une administration toutes les deux semaines selon l'évaluation clinique.
VYVGART a montré des résultats de sécurité cohérents avec les études cliniques précédentes, et 99 % des participants à l'essai ont poursuivi dans l'extension en ouvert. La décision finale de la Commission européenne concernant l'autorisation de mise sur le marché est attendue dans environ deux mois.
argenx hat eine positive CHMP-Empfehlung erhalten, die die Zulassung der Europäischen Kommission für VYVGART (Efgartigimod alfa) zur subkutanen Injektion zur Behandlung der chronisch entzündlichen demyelinisierenden Polyneuropathie (CIDP) empfiehlt. Die Behandlung stellt den ersten neuartigen Wirkmechanismus für CIDP seit über 30 Jahren dar.
Die Empfehlung basiert auf der ADHERE-Studie, die zeigte, dass 66,5 % der Patienten eine klinische Verbesserung zeigten. Die Studie erreichte ihren primären Endpunkt mit einer 61%igen Reduktion des Rückfallrisikos im Vergleich zu Placebo. Die Behandlung kann von Patienten, Betreuern oder medizinischem Fachpersonal verabreicht werden, mit einer anfänglichen wöchentlichen Dosierung, die je nach klinischer Bewertung auf alle zwei Wochen angepasst werden kann.
VYVGART zeigte konsistente Sicherheitsdaten im Einklang mit vorherigen klinischen Studien, und 99 % der Studienteilnehmer setzten die Behandlung in der offenen Verlängerung fort. Die endgültige Entscheidung der Europäischen Kommission zur Marktzulassung wird in etwa zwei Monaten erwartet.
- First-and-only targeted IgG Fc-antibody fragment treatment for CIDP
- 66.5% of patients showed clinical improvement in ADHERE trial
- 61% reduction in relapse risk versus placebo
- 99% trial participant retention rate in extension study
- Flexible self-administration option available
- Requires prior treatment with corticosteroids or immunoglobulins
- to adult patients only
Insights
VYVGART's CHMP recommendation for CIDP represents first novel treatment in 30 years, significantly expanding market potential and reinforcing argenx's autoimmune leadership.
The positive CHMP opinion for VYVGART in CIDP represents a significant commercial milestone for argenx. As the first novel mechanism for CIDP in over 30 years, VYVGART addresses a substantial unmet need in a therapeutic area that has seen little innovation despite the debilitating nature of the disease.
The ADHERE trial results are remarkably strong, showing a
The subcutaneous formulation offers crucial advantages for chronic administration, enabling at-home treatment by patients or caregivers. This represents a significant improvement over intravenous immunoglobulin (IVIG), which requires frequent hospital visits and lengthy infusions - particularly challenging for patients with mobility limitations from CIDP.
This indication expansion significantly increases VYVGART's commercial potential. While already approved for myasthenia gravis, adding CIDP substantially enlarges the addressable patient population and reinforces argenx's emerging leadership in autoimmune disorders.
The European Commission decision expected within two months provides a clear near-term catalyst. The consistently favorable safety profile and impressive
VYVGART brings transformative targeted therapy to CIDP patients with impressive 61% relapse reduction and patient-friendly administration, addressing significant unmet needs.
The CHMP positive opinion for VYVGART represents a potential paradigm shift in CIDP treatment. Current therapy options primarily involve broad immunosuppression with corticosteroids or the resource-intensive administration of immunoglobulins, both associated with significant limitations and side effects.
VYVGART's mechanism as a targeted IgG Fc-antibody fragment provides precision that current treatments lack. By selectively reducing pathogenic IgG antibodies through FcRn blockade, it addresses the underlying autoimmune pathology without comprehensive immune suppression. This targeted approach explains both the impressive efficacy and favorable safety profile.
The ADHERE trial data is particularly compelling as the largest CIDP study to date. The
The subcutaneous formulation with flexible dosing (weekly initially, potentially adjustable to biweekly) enables personalized treatment approaches. Self-administration capabilities significantly reduce treatment burden compared to current standards like IVIG, which require lengthy infusions in healthcare settings.
The extraordinary
- VYVGART® is first-and-only targeted IgG Fc-antibody fragment for CIDP
- First novel mechanism of action for CIDP treatment in more than 30 years
- CHMP positive opinion based on ADHERE data, the largest ever CIDP clinical trial
- European Commission (EC) decision on marketing authorization application (MAA) expected within approximately two months
April 28, 2025, 07:00 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 the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended European Commission (EC) approval of VYVGART® 1000mg (efgartigimod alfa) for subcutaneous (SC) injection as a monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins.
“Our mission is to develop innovative, targeted treatments for patients with rare and severe autoimmune diseases, who continue to face significant unmet needs. The positive CHMP opinion for VYVGART in CIDP brings us one step closer to providing patients across Europe with a transformational new treatment option that provides meaningful functional improvement,” said Luc Truyen M.D., Ph.D., Chief Medical Officer, argenx. “VYVGART is the first and only targeted IgG Fc-antibody fragment for CIDP and if approved, would mark the first treatment in Europe with a novel, precision mechanism of action for CIDP patients in 30 years.”
VYVGART for subcutaneous injection is available as a vial or prefilled syringe and can be administered by a patient, caregiver, or healthcare professional. Treatment is initiated with a weekly dose regimen and may be adjusted to every other week based on clinical evaluation.
The CHMP recommendation is based on positive results from the ADHERE clinical trial, the largest study of CIDP patients to date. In the ADHERE study,
"For the patient population represented by the European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN) and for those affected by CIDP, this is excellent news," said Jean-Philippe Plançon, President of EPODIN. "There are still considerable unmet medical needs in the management of CIDP, and the CHMP’s recommendation brings renewed hope for improved treatment options and quality of life."
The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC’s final decision on argenx’s CIDP application for subcutaneous VYVGART. The EC is expected to make a decision following CHMP recommendation and the decision will apply to all 27 European Union Member States, and also to Iceland, Norway and Liechtenstein. Currently, VYVGART is indicated as an add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
About ADHERE
The ADHERE trial was a multicenter, randomized, double-blind, placebo-controlled trial evaluating SC efgartigimod alfa for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE enrolled 322 adult patients with CIDP, 130 of whom were based in Europe, who were off treatment (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 SC efgartigimod alfa. 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 SC efgartigimod alfa 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 SC efgartigimod alfa.
About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. 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 worsen 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. There are an estimated 31,413 people living with CIDP in the European Union.
About Efgartigimod
Efgartigimod is an antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor and blocking the IgG recycling process. Efgartigimod is being investigated in several autoimmune diseases known to be mediated by disease-causing IgG antibodies, including neuromuscular disorders, blood disorders, and skin blistering diseases, in both an IV and SC formulation. SC efgartigimod is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology. In August 2022, efgartigimod received approval from the EC for IV administration as an add on to standard therapy for the treatment of adult patients with gMG who are AChR antibody positive.
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 and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, Instagram, Facebook, and YouTube.
Contacts
Media:
Kate Dion
kdion@argenx.com
Investors:
Alexandra Roy
aroy@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 “aim,” “are,” “believe,” “can,” “continue,” “expect,” “may,” and “will” and include statements argenx makes concerning the expected timing and decision of the EC regarding VYVGART for SC injection for CIDP treatment and the application of such decision; the potential for improved treatment options and quality of life; 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 the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements; the acceptance of its products and product candidates by its patients as safe, effective and cost-effective; the impact of governmental laws and regulations, including tariffs, export controls, sanctions and other regulations on its business; its reliance on third-party suppliers, service providers and manufacturers; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. 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.
FAQ
What are the clinical trial results for VYVGART in CIDP treatment?
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Who is eligible for VYVGART CIDP treatment in Europe?
