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SpringWorks Therapeutics (NYSE:SWTX) has secured conditional marketing authorization from the European Commission for EZMEKLY® (mirdametinib), a potential game-changer for both adult and pediatric patients (2 years and older) grappling with NF1-PN. This pivotal approval marks EZMEKLY as the first and only therapy sanctioned in the EU to combat this rare genetic disorder, impacting approximately 3 in 10,000 Europeans.
The green light from regulators stems from compelling Phase 2b ReNeu trial data, encompassing 114 patients. The results showcased an objective response rate of 41% in adult patients and a noteworthy 52% in pediatric cases. Across both groups, the median tumor volume reduction hovered around 41-42%, with nearly 90% of responders maintaining this progress for at least 12 months – underscoring a remarkable durability of response.
NF1, a condition affecting an estimated 135,000 people in the EU, often leads to the development of plexiform neurofibromas in 30-50% of cases. EZMEKLY’s novel availability in both capsule and dispersible tablet forms offers a ray of hope, providing a much-needed therapeutic avenue for a patient population previously starved of options.
SpringWorks Therapeutics (NYSE:SWTX) ha ottenuto l’autorizzazione condizionata alla commercializzazione dalla Commissione Europea per EZMEKLY® (mirdametinib) nel trattamento della NF1-PN sia negli adulti che nei pazienti pediatrici a partire dai 2 anni di età. Questo rappresenta un traguardo importante, poiché EZMEKLY diventa la prima e unica terapia approvata nell’UE per questa rara malattia genetica.
L’approvazione si basa sui risultati dello studio di Fase 2b ReNeu, che ha coinvolto 114 pazienti e ha dimostrato un’efficacia significativa con un tasso di risposta obiettiva del 41% negli adulti e del 52% nei bambini. La riduzione mediana del volume tumorale è stata di circa il 41-42% in entrambi i gruppi, con una durata della risposta di almeno 12 mesi in quasi il 90% dei pazienti responder.
La NF1 colpisce circa 3 persone su 10.000 nell’UE, con il 30-50% che sviluppa neurofibromi plessiformi. EZMEKLY sarà disponibile sia in capsule che in compresse dispersibili, offrendo una nuova opzione terapeutica per questa popolazione di pazienti finora poco servita.
SpringWorks Therapeutics (NYSE:SWTX) ha recibido la autorización condicional de comercialización por parte de la Comisión Europea para EZMEKLY® (mirdametinib) para tratar NF1-PN en pacientes adultos y pediátricos a partir de los 2 años. Esto representa un hito importante, ya que EZMEKLY se convierte en la primera y única terapia aprobada en la UE para este raro trastorno genético.
La aprobación se basa en los resultados del ensayo de fase 2b ReNeu con 114 pacientes, que mostraron una eficacia notable con una tasa de respuesta objetiva del 41% en adultos y 52% en niños. La reducción mediana del volumen tumoral fue aproximadamente del 41-42% en ambos grupos, con una durabilidad de la respuesta de al menos 12 meses en casi el 90% de los respondedores.
La NF1 afecta aproximadamente a 3 de cada 10,000 personas en la UE, con un 30-50% desarrollando neurofibromas plexiformes. EZMEKLY estará disponible en forma de cápsulas y tabletas dispersables, ofreciendo una nueva opción de tratamiento para esta población de pacientes previamente desatendida.
SpringWorks Therapeutics (NYSE:SWTX)는 유럽연합 집행위원회로부터 EZMEKLY® (mirdametinib)의 조건부 시판 허가를 받았습니다. 이 약은 2세 이상 성인 및 소아 환자의 NF1-PN 치료에 사용됩니다. 이는 EZMEKLY가 이 희귀 유전 질환에 대해 EU에서 최초이자 유일하게 승인된 치료제가 되는 중요한 이정표입니다.
승인은 114명의 환자를 대상으로 한 2b상 ReNeu 시험 결과에 기반하며, 성인에서 41%, 소아에서 52%의 객관적 반응률을 보이며 뛰어난 효능을 입증하였습니다. 두 그룹 모두에서 종양 부피가 약 41-42% 감소하였고, 반응 지속 기간은 반응자 중 거의 90%에서 최소 12개월 이상 유지되었습니다.
NF1은 EU 내 약 10,000명당 3명에게 영향을 미치며, 30-50%는 다발성 신경섬유종을 발병합니다. EZMEKLY는 캡슐과 분산 정제 형태로 제공되어, 이전에 치료 옵션이 부족했던 환자군에 새로운 치료 기회를 제공합니다.
SpringWorks Therapeutics (NYSE:SWTX) a reçu une autorisation de mise sur le marché conditionnelle de la Commission européenne pour EZMEKLY® (mirdametinib) afin de traiter la NF1-PN chez les patients adultes et pédiatriques âgés de 2 ans et plus. Cela représente une étape majeure, car EZMEKLY devient la première et unique thérapie approuvée dans l’UE pour cette maladie génétique rare.
Cette approbation repose sur les résultats de l’essai de phase 2b ReNeu impliquant 114 patients, qui ont démontré une efficacité impressionnante avec un taux de réponse objective de 41 % chez les adultes et 52 % chez les enfants. La réduction médiane du volume tumoral était d’environ 41-42 % dans les deux groupes, avec une durabilité de la réponse d’au moins 12 mois chez près de 90 % des répondeurs.
La NF1 touche environ 3 personnes sur 10 000 dans l’UE, dont 30 à 50 % développent des neurofibromes plexiformes. EZMEKLY sera disponible sous forme de capsules et de comprimés dispersibles, offrant une nouvelle option thérapeutique pour cette population de patients auparavant peu prise en charge.
SpringWorks Therapeutics (NYSE:SWTX) hat von der Europäischen Kommission eine bedingte Marktzulassung für EZMEKLY® (Mirdametinib) zur Behandlung von NF1-PN bei Erwachsenen und pädiatrischen Patienten ab 2 Jahren erhalten. Dies stellt einen bedeutenden Meilenstein dar, da EZMEKLY die erste und einzige in der EU zugelassene Therapie für diese seltene genetische Erkrankung ist.
Die Zulassung basiert auf den Ergebnissen der Phase-2b-Studie ReNeu mit 114 Patienten, die eine beeindruckende Wirksamkeit mit einer objektiven Ansprechrate von 41 % bei Erwachsenen und 52 % bei Kindern zeigte. Die mediane Tumorvolumenreduktion betrug in beiden Gruppen etwa 41-42 %, wobei die Ansprechdauer bei fast 90 % der Ansprechenden mindestens 12 Monate betrug.
NF1 betrifft etwa 3 von 10.000 Menschen in der EU, wobei 30-50 % Plexiforme Neurofibrome entwickeln. EZMEKLY wird sowohl in Kapselform als auch als dispersible Tablette erhältlich sein und bietet eine neue Behandlungsoption für diese bisher unterversorgte Patientengruppe.
Positive
- First and only EU-approved therapy for both adults and children with NF1-PN
- Strong efficacy with 41% response rate in adults and 52% in children
- Significant tumor volume reduction of ~41-42% in both adult and pediatric patients
- High durability of response with ~90% maintaining response for at least 12 months
- Available in both capsule and dispersible tablet forms for improved accessibility
Negative
- High frequency of adverse reactions (83% dermatitis acneiform in adults)
- Conditional approval status indicates additional confirmatory data may be required
- Multiple common side effects including diarrhea, nausea, and increased blood creatine phosphokinase
Insights
EC approval for EZMEKLY as first-in-EU therapy for adult/pediatric NF1-PN marks significant commercial opportunity for SpringWorks.
The European Commission’s conditional approval of EZMEKLY (mirdametinib) represents a significant regulatory milestone for SpringWorks Therapeutics. This approval establishes EZMEKLY as the first and only therapy approved in the European Union for both adults and children with NF1-PN, a rare genetic disorder affecting approximately 135,000 people in the EU.
The clinical data from the Phase 2b ReNeu trial shows impressive efficacy with objective response rates of 41% in adults and 52% in children. The durability of these responses is particularly notable, with 88% of adult responders and 90% of pediatric responders maintaining responses for at least 12 months. The median tumor volume reduction of approximately 40% in both populations further validates the drug’s efficacy.
From a commercial perspective, this approval addresses a clear unmet need in a rare disease with limited treatment options. With 30-50% of NF1 patients developing plexiform neurofibromas and 85% of these tumors considered inoperable, EZMEKLY has a well-defined target patient population. The availability in multiple formulations, including a dispersible tablet, should enhance patient compliance and market penetration.
The manageable safety profile with primarily Grade 1-2 adverse events supports long-term treatment feasibility, critical for a chronic condition. While specific commercial projections weren’t provided, this approval unlocks access to the EU’s 135,000 NF1 patients, representing a significant market opportunity for SpringWorks as they expand their global commercial footprint beyond the US market.
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