#Australia #Amsterdam #Norgine #CISPLATIN #PEDMARQSI

Norgine's Breakthrough in Pediatric Oncology: TGA Registration of PEDMARQSI®

On May 5, 2026, Norgine, a prominent European pharmaceutical firm, proudly announced that the Therapeutic Goods Administration (TGA) in Australia has registered PEDMARQSI® (sodium thiosulfate anhydrous). This significant milestone represents a critical advancement in pediatric oncology, specifically targeting the prevention of cisplatin-induced hearing loss in children aged one month to less than 18 years with localized, non-metastatic solid tumors.

Cisplatin, an essential chemotherapeutic agent, is widely utilized in treating solid tumors in pediatric patients. However, it is accompanied by substantial risks, including irreversible hearing loss, which can lead to severe consequences for children, affecting their speech, language development, educational pursuits, social interactions, and overall quality of life. PEDMARQSI® stands out as the first and only registered treatment in Australia designed to avert this distressing side effect of cisplatin chemotherapy.

"The TGA registration of PEDMARQSI® marks an important milestone for children, families, and healthcare professionals in Australia," commented Gus Rudolph, General Manager of Norgine, Australia. "The approval provides an option specifically developed to help prevent this complication in eligible children receiving cisplatin chemotherapy."

Dr. David Gillen, the Chief Medical Officer of Norgine, emphasized the clinical importance of this development, noting that cisplatin-induced hearing loss is a recognized complication with substantial long-term implications for children's communication, learning, and quality of life. He stated, "The availability of an approved preventive option represents a meaningful development in the supportive care of pediatric oncology patients."

The TGA's decision to approve PEDMARQSI® was based on data from two open-label, randomized Phase 3 clinical trials—SIOPEL 6 and COG ACCL0431, which established the effectiveness and safety of the treatment.

In addition to its recent approval in Australia, PEDMARQSI® has also received marketing authorization in other regions, including the Pediatric Use Marketing Authorisation (PUMA) from the European Medicines Agency in May 2023, as well as approvals from the UK and Swissmedic in March 2026. This progressive regulatory success illustrates Norgine's commitment to navigating the complexities of bringing rare and specialist medicines to market.

While the drug has not yet been included on the Pharmaceutical Benefits Scheme (PBS), Norgine remains dedicated to collaborating with relevant stakeholders to ensure access for eligible patients throughout Australia. The company acknowledges the importance of this treatment not only for enhancing the healthcare landscape but also for the emotional and physical well-being of children undergoing potentially life-saving cisplatin therapy.

PEDMARQSI® is a specialized formulation of sodium thiosulfate anhydrous, meticulously crafted for preventing cisplatin-induced hearing loss. It is worth noting that safety information regarding possible risks, side effects, and precautions—including allergic reactions, nausea, and electrolyte imbalances—should be reviewed carefully by healthcare professionals and patients alike.

As an innovative EU-based pharmaceutical company with around 1,500 employees and approximately $650 million in annual sales, Norgine is on a mission to deliver transformative medicines addressing unmet medical needs. From commonplace ailments to severe health challenges, the organization harnesses its expertise to expedite the development and distribution of essential pharmaceuticals across Europe, Australia, and New Zealand.

This remarkable achievement highlights Norgine's dedication to ensuring that every scientific breakthrough reaches those in need, emphasizing the importance of innovation in healthcare to transform lives, one patient at a time.