#Amsterdam #Switzerland #Norgine #CISPLATIN #PEDMARQSI

Norgine's PEDMARQSI® Gains Approval to Prevent Hearing Loss

Norgine B.V., a prominent European specialty pharmaceutical company, recently celebrated a significant milestone with the announcement of Swissmedic's approval of PEDMARQSI®. This medication, a formulation of sodium thiosulfate, has been recognized as the first and only treatment in Switzerland aimed specifically at preventing cisplatin-induced hearing loss in pediatric patients aged between one month and eighteen years suffering from localized, non-metastatic solid tumors.

The approval signifies a groundbreaking advancement in addressing urgent medical needs, particularly for children undergoing chemotherapy with cisplatin, a drug effective in treating various pediatric cancers but notorious for its potential to cause irreversible bilateral hearing loss. The adverse effects can commence with the very first dose and may worsen over time, adversely affecting a child's speech and language development, educational progress, psychosocial growth, and overall quality of life.

Rüdiger Merkel, Managing Director of Norgine for the DACH region, remarked, "The authorization from Swissmedic for PEDMARQSI® represents a critical development for children, families, and healthcare professionals in Switzerland. Previously, there were no approved pharmacological options available to prevent this complication associated with cisplatin treatment. The significant impact of hearing loss on young patients makes this authorization crucial, as it grants access to a treatment specifically developed to address this unmet medical need."

The approval is grounded in data from two pivotal phase 3 randomized and open-label trials: SIOPEL 6 and COG ACCL0431. These studies demonstrated that administering sodium thiosulfate alongside cisplatin resulted in approximately a 50% reduction in the occurrence of cisplatin-induced ototoxicity compared to the administration of cisplatin alone, while preserving the chemotherapy's effectiveness.

In May 2023, the European Medicines Agency (EMA) had previously granted PEDMARQSI® a pediatric use marketing authorization, along with a national marketing authorization in the United Kingdom. These regulatory approvals highlight Norgine’s proficiency in navigating the diverse European regulatory frameworks for specialized medications targeted at rare conditions.

About PEDMARQSI®

PEDMARQSI® is a novel formulation of anhydrous sodium thiosulfate designed specifically to prevent cisplatin-induced hearing loss in pediatric patients. It is unique in the sense that it is the first preventive treatment tailored for addressing this type of ototoxicity. The marketing authorizations are based on security and efficacy data obtained from pivotal phase 3 studies, which have robustly indicated that sodium thiosulfate significantly reduces the risk of hearing loss associated with cisplatin chemotherapy.

The clinical trials report favorable tolerance profiles, with nausea and vomiting being the most commonly observed side effects. More severe adverse effects reported include infections, anemia, and neutropenia, classified under grade 3-4 complications.

About Norgine

Norgine is a mid-sized European pharmaceutical company that employs approximately 1,500 individuals and generates an annual revenue of around 650 million dollars. At the heart of Norgine's mission is innovation, aiming to deliver life-changing medication options. Whether addressing common conditions such as constipation or tackling severe and rare diseases like pediatric cancer, Norgine focuses on meeting unsatisfied medical needs, believing that every scientific advancement deserves to reach the patients in need.

Using its innovative development, marketing, and manufacturing capabilities, alongside strategic partnerships, Norgine is committed to delivering on this complex mission. Coupled with its rich history and significant regional expertise, Norgine strives to accelerate and expand access to transformative medications across Europe, Australia, and New Zealand. The company is driven by the trust placed in them by healthcare professionals and patients alike, pledging to provide innovations that transform lives, one patient at a time.