Norgine Celebrates TGA Approval of PEDMARQSI® in Australia for Pediatric Hearing Loss Prevention

Norgine’s Major Milestone with TGA Approval of PEDMARQSI®

Norgine, a prominent European pharmaceutical company, recently celebrated a significant achievement with the registration of its drug PEDMARQSI® (anhydrous sodium thiosulfate) by the Australian Therapeutic Goods Administration (TGA). This groundbreaking drug is aimed at preventing ototoxicity induced by cisplatin chemotherapy in pediatric patients aged from one month to under 18 years, specifically for those suffering from localized, non-metastatic solid tumors.

The Significance of PEDMARQSI®

Cisplatin is a widely-used chemotherapeutic agent known for its effectiveness yet carries a considerable risk of causing permanent hearing loss, particularly in young patients. Hearing loss during childhood can lead to numerous challenges, including adverse effects on speech and language development, education, social interactions, and overall quality of life. PEDMARQSI® emerges as the first and only registered preventive treatment in Australia for this specific complication in children undergoing treatment with cisplatin.

Gus Rudolph, Managing Director of Norgine Australia, expressed, "The registration of PEDMARQSI® represents a pivotal moment for children, families, and healthcare professionals in Australia. The ototoxicity resulting from cisplatin can have lifelong consequences, and this approval provides a much-needed option tailored explicitly to prevent this complication for eligible pediatric patients undergoing chemotherapy with cisplatin. We are delighted to reach this regulatory milestone and remain committed to collaborating with relevant stakeholders to ensure accessible treatment for eligible patients in Australia."

Clinical Trial Success

The TGA's approval of PEDMARQSI® is grounded in data derived from two pivotal phase 3 clinical trials, the SIOPEL 6 and COG ACCL0431. These trials highlight the substantial need for effective preventive measures against cisplatin-induced hearing loss, a well-documented but often irreversible complication of pediatric cancer treatment. Dr. David Gillen, Chief Medical Officer of Norgine, added, "Addressing the risk of hearing loss proactively is crucial for the treatment of children receiving cisplatin. The availability of an approved preventive option is a significant advancement in the comprehensive care of pediatric oncology patients."

Regulatory Pathway and Recognitions

The marketing authorization of PEDMARQSI® in Australia complements previous approvals in other jurisdictions, including the Pediatric Use Marketing Authorization (PUMA) granted by the European Medicines Agency in May 2023, a national marketing authorization in the United Kingdom, and approval from Swissmedic in March 2026. These accomplishments underscore Norgine's ability to navigate regulatory pathways for rare and specialized medications effectively.

Currently, PEDMARQSI® is not included in the Pharmaceutical Benefits Scheme (PBS). For comprehensive information regarding safety, risks, side effects, and precautions related to PEDMARQSI®, including potential allergic reactions, nausea, vomiting, and electrolyte imbalances, patients and healthcare professionals are advised to refer to the Consumer Medicines Information (CMI).

About PEDMARQSI®

PEDMARQSI® is specifically formulated to prevent cisplatin-induced hearing loss in pediatric patients with localized solid tumors. It is the primary preventive treatment available for this demographic at risk of complications from cisplatin therapy. The marketing approvals for PEDMARQSI® were based on safety and efficacy data derived from the two aforementioned phase III trials.

For more information about PEDMARQSI®, including the CMI, visit the TGA website.

About Norgine

Norgine is a medium-sized European pharmaceutical company employing approximately 1,500 people and generating around €650 million in annual sales. The company's mission, driven by innovation, is to deliver life-transforming medications. Norgine focuses on unmet medical needs, producing innovative solutions from common conditions like constipation to severe rare diseases such as pediatric cancer, ensuring that every scientific advancement reaches the patients who need them.

Leveraging its innovative development, marketing, and manufacturing capabilities, together with strategic partnerships, Norgine tackles complex challenges and enhances the reach of transformative medicines across Europe, Australia, and New Zealand. The trust that healthcare professionals and patients place in Norgine underpins its commitment to delivering innovations that change lives, one patient at a time. The NORGINE and sail logo are trademarks of the Norgine group of companies.