Norgine Receives Swiss Approval for PEDMARQSI, First Treatment to Prevent Cisplatin-Induced Hearing Loss in Children
核心洞察
Swissmedic (搜索) has approved PEDMARQSI (sodium thiosulfate solution) as the first and only treatment authorized in Switzerland for preventing cisplatin-induced hearing loss (搜索) in pediatric cancer (搜索) patients aged 1 month to 18 years.
Clinical trials demonstrated approximately 50% reduction in cisplatin-induced ototoxicity (搜索) when sodium thiosulfate was administered alongside cisplatin compared to cisplatin alone, while maintaining chemotherapy efficacy.
The approval addresses a significant unmet medical need, as cisplatin-induced hearing loss (搜索) can begin with the first dose and have lifelong impacts on children's speech, language development, and quality of life.
Norgine B.V. announced that Swissmedic (搜索) has granted marketing authorization for PEDMARQSI (sodium thiosulfate solution for infusion), marking a significant milestone in pediatric oncology care. The approval makes PEDMARQSI the first and only pharmacological treatment specifically authorized in Switzerland for preventing cisplatin-induced ototoxicity (搜索) in patients aged 1 month to less than 18 years with localized, non-metastatic solid tumors (搜索).
Addressing Critical Unmet Medical Need
Cisplatin remains a cornerstone of pediatric cancer (搜索) treatment but carries a high risk of irreversible, bilateral hearing loss (搜索) that can begin with the first dose and progress over time. This ototoxicity can have profound and lasting impacts on a child's speech and language acquisition, education, psychosocial development, and overall quality of life.
"The Swissmedic (搜索) approval of PEDMARQSI represents an important milestone for children, families and healthcare professionals in Switzerland, where no approved pharmacological option has previously been available to help prevent this complication of cisplatin treatment," said Rüdiger Merkel, General Manager, DACH, Norgine. "Hearing loss (搜索) can have a lifelong impact on young patients, and this approval supports access to a treatment developed specifically to address this unmet medical need."
Clinical Evidence Supporting Approval
The Swissmedic (搜索) approval is based on clinical evidence from two open-label, randomized Phase 3 trials: SIOPEL 6 (pivotal) and the Clinical Oncology Group (COG) ACCL0431. Across these studies, treatment with sodium thiosulfate administered alongside cisplatin resulted in approximately a 50% reduction in the occurrence of cisplatin-induced ototoxicity (搜索) compared with cisplatin alone, while maintaining the efficacy of chemotherapy.
The trials demonstrated a favorable tolerability profile, with nausea and vomiting being the most commonly reported adverse events, and infection, anemia, and neutropenia being the most common grade 3-4 adverse events.
Novel Formulation and Regulatory Success
PEDMARQSI is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss (搜索) in the target patient population. The drug has previously been granted pediatric use marketing authorization (PUMA) by the European Medicines Agency (搜索) (EMA) in May 2023 in the European Union and received national marketing authorization in the United Kingdom.
These approvals reflect Norgine's ability to successfully navigate diverse European regulatory pathways for rare and specialist medicines. The company is a mid-sized EU-based pharmaceutical company with 1,500 employees, generating approximately $650 million in annual sales, focusing on delivering medicines that address unmet medical needs from common conditions to rare and severe diseases such as childhood cancer.