#Australia #Amsterdam #Hearing Loss #Norgine #PEDMARQSI

Norgine Celebrates TGA Approval for PEDMARQSI®

In a significant advancement for pediatric healthcare, Norgine, a leading European pharmaceutical company, has announced the registration of PEDMARQSI® (anhydrous sodium thiosulfate) by the Therapeutic Goods Administration (TGA) in Australia. This innovative therapy is designed specifically for preventing hearing loss induced by cisplatin chemotherapy in children aged from one month to under 18 years diagnosed with localized, non-metastatic solid tumors.

Cisplatin is widely recognized as a pivotal chemotherapeutic agent for treating various pediatric cancers. However, its administration comes with the daunting risk of permanent hearing loss, a complication that can have profound and lasting repercussions on a child’s ability to communicate, learn, and maintain a quality social life. Children affected by such hearing loss may face significant challenges in speech and language development, education, and overall well-being.

PEDMARQSI® is not only the first but also the only treatment approved in Australia aimed at preventing cisplatin-induced ototoxicity in pediatric patients. According to Gus Rudolph, Managing Director of Norgine Australia, “The registration of PEDMARQSI® marks a crucial milestone for children, families, and healthcare professionals in Australia. The risk of hearing loss associated with cisplatin can result in lifelong consequences for young patients, and this approval provides a dedicated option to help prevent this complication for eligible children undergoing treatment.”

The TGA's approval of PEDMARQSI® is backed by data from two pivotal Phase 3 clinical trials, namely SIOPEL 6 and COG ACCL0431. Dr. David Gillen, Medical Director at Norgine, emphasized the critical nature of addressing this risk proactively in the treatment of children receiving cisplatin. “The availability of an approved preventive option represents a significant development in supportive care for pediatric oncology patients,” he stated.

This registration not only enhances treatment possibilities in Australia but also complements existing approvals in other regions, including a Pediatric Use Marketing Authorization (PUMA) granted by the European Medicines Agency in May 2023, as well as national marketing authorization in the UK and approval from Swissmedic in March 2026. These achievements underscore Norgine's capability to navigate the regulatory pathways for rare and specialized medicines.

While PEDMARQSI® has recently received TGA approval, it is currently not listed under the Pharmaceutical Benefits Scheme (PBS). Comprehensive information regarding the risks, side effects, and precautions associated with PEDMARQSI® can be found in the Consumer Medicines Information (CMI).

About PEDMARQSI®

PEDMARQSI® is a specifically formulated therapy utilizing anhydrous sodium thiosulfate for preventing cisplatin-induced hearing loss in pediatric patients. It stands as the first and only preventive treatment developed for this specific complication. Its efficacy and safety have been evaluated through robust clinical trials, signifying a leap forward in the management of pediatric patients at risk of ototoxicity due to chemotherapy.

About Norgine

Norgine is a mid-sized European pharmaceutical company employing approximately 1,500 staff and achieving an annual revenue of around $650 million. Innovation drives Norgine’s mission: to provide life-changing medications. Whether addressing common ailments or tackling serious conditions like pediatric cancer, Norgine is committed to fulfilling unmet medical needs, with a focus on bringing scientific advancements to patients who need them the most.

With strong development, marketing, and manufacturing capabilities, coupled with strategic partnerships, Norgine aims to accelerate and increase the availability of vital medications across Europe, Australia, and New Zealand.