#Australia #Amsterdam #Norgine #CISPLATIN #PEDMARQSI®

Norgine Celebrates TGA Approval for PEDMARQSI®

Norgine, a prominent specialty pharmaceutical company based in Europe, is pleased to announce a major breakthrough in pediatric oncology. The Australian Therapeutic Goods Administration (TGA) has officially registered PEDMARQSI® (sodium thiosulfate anhydrous) for the prevention of hearing loss caused by cisplatin chemotherapy in young patients aged 1 month to under 18 years, who are battling localized, non-metastatic solid tumors. This marks a crucial step in addressing a significant unmet medical need in the field of child healthcare.

Cisplatin is a widely utilized chemotherapeutic agent for treating pediatric tumors, but it poses a serious risk of ototoxicity, leading to irreversible hearing loss. Such losses during childhood can have deep and lasting effects, impacting not only speech and language development but also overall education and quality of life. Given this context, the introduction of PEDMARQSI® offers hope for many families and health professionals who have been looking for effective solutions in this area.

Gus Rudolph, General Manager of Norgine in Australia, commented, "The TGA registration of PEDMARQSI® is a pivotal moment for families and healthcare providers. The potential for hearing loss from cisplatin can have lifelong implications for such young patients, and this approval introduces a dedicated treatment option to mitigate that risk. We are excited to achieve this regulatory milestone and will work closely with stakeholders to facilitate access for patients across Australia."

The approval comes on the back of robust clinical data derived from two Phase 3 randomized clinical trials: SIOPEL 6 and COG ACCL0431, which collectively underscore the efficacy and safety of PEDMARQSI® in this vulnerable population. As Dr. David Gillen, Norgine's Chief Medical Officer noted, "The recognition of cisplatin-induced hearing loss as a common and serious complication in pediatric cancer treatments highlights the need for preventive measures. The availability of PEDMARQSI® signifies a vital advancement in supportive care for young oncology patients."

With the TGA's decision, PEDMARQSI® now joins the ranks of other global marketing authorizations, including the European Medicines Agency's PUMA granted in May 2023 and approvals from the UK's Medicines and Healthcare products Regulatory Agency and Swissmedic in 2026. This demonstrates Norgine's dedication to navigating complex regulatory frameworks to facilitate access to rare and vital medicines.

Currently, PEDMARQSI® is not listed under the Pharmaceutical Benefits Scheme (PBS) in Australia, and it's essential for patients and caregivers to consult the PEDMARQSI® Consumer Medicines Information (CMI) for comprehensive safety guidelines, including potential side effects such as allergic reactions and electrolyte imbalances.

About PEDMARQSI®

PEDMARQSI® is specifically formulated to prevent cisplatin-induced hearing loss in young patients with localized, non-metastatic solid tumors. As the only preventive treatment of its kind available in Australia, it addresses a critical gap in pediatric cancer care. The marketing authorizations for PEDMARQSI® rest on substantial safety and effectiveness data from the SIOPEL 6 and COG ACCL0431 trials.

For more details about PEDMARQSI®, please refer to its Consumer Medicines Information or visit the TGA website.

About Norgine

Norgine operates as a mid-sized pharmaceutical entity with approximately 1,500 employees and $650 million in annual sales. The company prides itself on innovation and its commitment to meeting urgent medical needs, addressing conditions ranging from common illnesses to rare diseases like childhood cancers. Through strategic partnerships and a focus on navigating the complexities of regulatory pathways, Norgine aims to expand the reach of transformative medicines across Europe, Australia, and New Zealand, always guided by the trust placed in them by healthcare professionals and patients alike.