AMSTERDAM, The Netherlands – May 6, 2026
Norgine has announced that the Australian Therapeutic Goods Administration (TGA) has granted registration for PEDMARQSI® (sodium thiosulfate anhydrous) for the prevention of cisplatin-induced ototoxicity in paediatric patients aged 1 month to under 18 years with localised, non-metastatic solid tumours. The approval marks a significant regulatory milestone as PEDMARQSI® becomes the first and only approved treatment in Australia specifically indicated to prevent hearing loss caused by cisplatin chemotherapy in children. The decision addresses a major unmet medical need in paediatric oncology, where permanent hearing damage remains one of the most serious long-term side effects associated with cisplatin-based cancer treatment.
First Approved Preventive Therapy for Cisplatin Hearing Loss
Cisplatin is widely used in paediatric oncology for the treatment of solid tumours due to its strong anti-cancer activity, but the therapy carries a substantial risk of irreversible hearing loss. Ototoxicity caused by cisplatin can significantly impact a child’s speech development, learning ability, communication skills, social interaction, and overall quality of life. PEDMARQSI® was specifically developed to reduce this risk and represents a major advancement in supportive cancer care for children undergoing chemotherapy.
According to Norgine, the TGA registration provides healthcare professionals in Australia with a dedicated preventive option designed to protect hearing while maintaining access to effective cancer treatment. The approval is particularly important because existing management strategies for chemotherapy-related hearing damage have historically been limited and largely supportive rather than preventive.
Phase 3 Trial Data Supported TGA Registration
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The Australian approval was supported by clinical data from two open-label, randomized Phase III trials — SIOPEL 6 and COG ACCL0431 — which evaluated the safety and efficacy of PEDMARQSI® in reducing cisplatin-induced hearing loss in paediatric cancer patients. The studies demonstrated clinically meaningful hearing protection benefits in children receiving cisplatin chemotherapy for localized solid tumours.
Norgine highlighted that the availability of a preventive therapy represents an important development in paediatric supportive oncology care, where long-term treatment complications can continue affecting patients well beyond cancer survival. Dr. David Gillen, Chief Medical Officer at Norgine, stated that proactively addressing chemotherapy-related hearing loss is increasingly recognized as a critical component of comprehensive paediatric cancer management.
Global Regulatory Expansion Strengthens PEDMARQSI Position
The TGA registration further expands PEDMARQSI®’s international regulatory footprint following earlier approvals from the European Medicines Agency (EMA), the United Kingdom, and Swissmedic in Switzerland. The growing number of regulatory authorizations highlights increasing recognition of the clinical importance of preventing cisplatin-related hearing loss in children.
Norgine noted that the approval also demonstrates the company’s ability to navigate regulatory pathways for specialized and rare-disease-focused medicines across multiple global markets. However, the company confirmed that PEDMARQSI® is not currently listed on Australia’s Pharmaceutical Benefits Scheme (PBS), meaning reimbursement discussions and patient access pathways may still evolve in the future.
Expanding Focus on Rare and Supportive Care Medicines
Norgine continues to position itself as a specialty pharmaceutical company focused on addressing unmet medical needs across both common and rare diseases. Beyond paediatric oncology supportive care, the company maintains operations across Europe, Australia, and New Zealand with a portfolio spanning gastrointestinal, oncology, and specialty therapeutics.
PEDMARQSI® reflects growing pharmaceutical industry interest in therapies that improve long-term treatment outcomes and quality of life for cancer survivors, particularly in paediatric populations where treatment-related complications can have lifelong consequences. With increasing emphasis on survivorship care in oncology, preventive supportive therapies such as PEDMARQSI® are expected to play an increasingly important role in comprehensive cancer treatment strategies.
Source: Norgine press release
