Norgine Celebrates TGA Approval of PEDMARQSI® for Children in Australia

Norgine's Vision for Pediatric Care

Norgine, a prominent European pharmaceutical company, proudly announces that the Therapeutic Goods Administration (TGA) in Australia has granted approval for PEDMARQSI®, a formulation of anhydrous sodium thiosulfate. This significant development marks a pivotal moment in the realm of pediatric oncology, focusing on preventing cisplatin-induced ototoxicity in children. In particular, this treatment applies to young patients aged from one month to less than 18 years with localized, non-metastatic solid tumors.

Addressing a Critical Medical Need

Cisplatin is an integral chemotherapy agent used widely among children with cancer, particularly those having solid tumors. However, its usage poses a substantial risk of permanent hearing loss, which can profoundly impact various facets of a child’s life, including their speech development, educational journey, social interactions, and overall quality of life. Importantly, PEDMARQSI® emerges as the first and only preventive treatment authorized in Australia specifically designed for this vulnerable group of patients.

According to Gus Rudolph, General Manager of Norgine Australia, “The TGA approval of PEDMARQSI® is a monumental milestone for children, families, and healthcare professionals in Australia. The risk of hearing loss due to cisplatin can have lifelong consequences for young patients. This approval provides an option specifically designed to prevent this complication in children undergoing cisplatin chemotherapy.”

Evidence-Based Approval Process

The TGA’s endorsement of PEDMARQSI® is grounded in robust evidence derived from two pivotal Phase 3 clinical trials, namely SIOPEL 6 and COG ACCL0431. As Dr. David Gillen, Norgine’s Chief Medical Officer, states, “Cisplatin-induced hearing loss is an acknowledged and often irreversible complication of pediatric cancer treatment, having significant long-term implications for communication, learning, and overall quality of life. Clinically, being able to proactively address this risk is an essential consideration in caring for children treated with cisplatin.”

Dr. Gillen highlights the significance of having an approved preventive option as it represents a crucial advancement in supporting pediatric oncology patients.

Expansion of Access to PEDMARQSI®

The market approval of PEDMARQSI® in Australia follows previous approvals in other regions, including a pediatric use authorization from the European Medicines Agency in May 2023, and national approval in the United Kingdom. Additionally, Swissmedic granted approval in March 2026.

This progression underscores Norgine’s capacity to navigate regulatory pathways for specialized and rare pharmaceuticals effectively. While PEDMARQSI® is not yet included in Australia's Pharmaceutical Benefits Scheme, the company continues striving to ensure access for eligible patients.

About PEDMARQSI®

PEDMARQSI® is formulated specifically to prevent hearing loss induced by cisplatin in patients aged from one month to less than 18 years with localized, non-metastatic solid tumors. This treatment is rooted in safety and efficacy data from previously conducted Phase 3 studies, emphasizing Norgine's commitment to addressing unmet medical needs.

For complete safety details, side effects, and precautions, including allergy risks and electrolyte imbalances, patients are encouraged to refer to the PEDMARQSI® Consumer Medicines Information (CMI).

Norgine: A Legacy of Innovation

Norgine is a mid-sized pharmaceutical company headquartered in the EU, employing roughly 1,500 staff members and generating an annual revenue of around 650 million USD. Dedicated to innovation, Norgine is focused on delivering medicines that transform lives, addressing both common and rare diseases. Through strategic partnerships and leveraging a thorough understanding of regional markets, Norgine accelerates the availability of life-changing medications across Europe, Australia, and New Zealand. The company prides itself on earning the trust of healthcare professionals and patients alike, remaining committed to developing innovations that positively influence the lives of individuals in need.

For more information on PEDMARQSI®, individuals can visit the TGA website or refer to the consumer medicine information link here.