Pharma firm Norgine B.V. has announced that Swissmedic has granted marketing authorisation for PEDMARQSI (sodium thiosulfate solution for infusion) indicated for the prevention of cisplatin induced ototoxicity (hearing loss) in patients aged 1 month to <18 years with localised, non-metastatic solid tumours.
PEDMARQSI is the first and only pharmacological treatment specifically developed to prevent cisplatin induced hearing loss in children.
While cisplatin remains a cornerstone of paediatric cancer treatment, it is associated with a high risk of irreversible, bilateral hearing loss that can begin with the first dose and progress over time. Hearing loss can have a profound and lasting impact on a child’s speech and language acquisition, education, psychosocial development and overall quality of life
Rüdiger Merkel, general manager, DACH, Norgine, said: “The Swissmedic 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. Hearing loss can have a lifelong impact on young patients, and this approval supports access to a treatment developed specifically to address this unmet medical need.”
The Swissmedic approval is based on clinical evidence from two open label, randomised Phase 3 trials. Across these studies, treatment with sodium thiosulfate administered alongside cisplatin resulted in approximately a 50% reduction in the occurrence of cisplatin-induced ototoxicity compared with cisplatin alone, while maintaining the efficacy of chemotherapy.2,3
PEDMARQSI has previously been granted paediatric use marketing authorisation (PUMA) by the European Medicines Agency (EMA) in May 2023 in the European Union and a national marketing authorisation in the United Kingdom.4,5 Together, these approvals reflect Norgine’s ability to successfully navigate diverse European regulatory pathways for rare and specialist medicines.
PEDMARQSI is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin induced hearing loss in patients aged 1 month to <18 years with localised, nonmetastatic solid tumours. It is the first and only preventative treatment developed for cisplatin induced ototoxicity in this patient population.1
Marketing authorisations for PEDMARQSI were based on safety and efficacy data from two open label, randomised Phase III trials: SIOPEL 6 (pivotal) and Clinical Oncology Group (COG) ACCL0431.2,3 Across these studies, sodium thiosulfate significantly reduced the risk of hearing loss associated with cisplatin chemotherapy. The trials demonstrated a favourable tolerability profile, with nausea and vomiting being the most commonly reported adverse events, and infection, anaemia and neutropenia being the most common grade 3–4 adverse events.
Norgine is a mid-sized EU-based pharmaceutical company with 1,500 employees, generating approximately $650 million in annual sales.