Norgine Receives Approval from Swissmedic for PEDMARQSI® to Combat Hearing Loss in Children

Norgine's Landmark Approval for PEDMARQSI®

In a groundbreaking announcement, Norgine B.V., a prominent European specialty pharmaceutical company, revealed that Swissmedic has granted marketing authorization for its innovative product, PEDMARQSI®. This solution, based on sodium thiosulfate for infusion, is the first and only treatment approved in Switzerland aimed at preventing cisplatin-induced ototoxicity (hearing loss) in children. Specifically, it is designed for patients aged from 1 month to under 18 years with localized, non-metastatic solid tumors.

The approval from Swissmedic comes as a significant milestone in addressing an urgent medical need. Children undergoing cisplatin chemotherapy, a standard treatment for various pediatric cancers, face a high risk of severe, irreversible hearing loss that can start with the very first dose and worsen over time. Such hearing loss can dramatically affect a child's speech development, educational progress, psychosocial development, and overall quality of life. Within this context, the introduction of PEDMARQSI® brings hope to families and healthcare professionals alike.

Rüdiger Merkel, the CEO of Norgine for the DACH region, emphasized the importance of this approval, stating, “The authorization of PEDMARQSI® by Swissmedic is a monumental step forward for children, families, and healthcare providers in Switzerland, where no pharmacological options existed to prevent complications arising from cisplatin treatment. Hearing loss can have lifelong repercussions for young patients, and this approval paves the way for a treatment specifically targeting this unmet medical need.”

The approval of PEDMARQSI® is founded on data from two pivotal phase 3, randomized, open-label trials: the SIOPEL 6 study and the ACCL0431 from the Clinical Oncology Group (COG). Results from these trials demonstrated approximately a 50% reduction in the frequency of cisplatin-induced ototoxicity in patients who received sodium thiosulfate alongside the chemotherapy compared to those treated with cisplatin alone. Remarkably, this treatment maintained the effectiveness of the chemotherapy regimen.

Previously, PEDMARQSI® had secured pediatric use marketing authorization (PUMA) from the European Medicines Agency (EMA) in May 2023, as well as a national marketing authorization in the United Kingdom. These achievements underscore Norgine's ability to successfully navigate diverse European regulatory pathways for rare and specialized drugs.

About PEDMARQSI®

PEDMARQSI® is an innovative formulation of anhydrous sodium thiosulfate, meticulously developed and manufactured to prevent cisplatin-induced hearing loss in pediatric patients aged 1 month to under 18 years with localized non-metastatic tumors. It stands as the first and only preventative treatment specifically targeting this form of ototoxicity in this vulnerable patient group. Based on robust safety and efficacy evidence from the previously mentioned phase III trials, PEDMARQSI® has been shown to significantly mitigate the risk of hearing loss linked to cisplatin chemotherapy. Common adverse effects observed during the trials included nausea and vomiting, with infections, anemia, and neutropenia being the most frequently reported grade 3-4 adverse events.

About Norgine

Norgine is a mid-sized pharmaceutical company headquartered in the EU, employing around 1,500 people and achieving annual sales of approximately $650 million. The company is dedicated to transforming lives through innovative medicines. They focus on unmet medical needs across a range of conditions, from common issues like constipation to severe, rare diseases such as pediatric cancer. Leveraging their innovative development, marketing, and manufacturing capabilities, along with strategic partnerships, Norgine strives to tackle complex challenges and expedite the delivery of life-changing medicines across Europe, Australia, and New Zealand.

Guided by the trust placed in them by healthcare professionals and patients alike, Norgine remains committed to spearheading innovations that transform lives, one patient at a time.