#Australia #Amsterdam #Norgine #PEDMARQSI #TGA Approval

Norgine Celebrates TGA Approval of PEDMARQSI®

Introduction

Norgine, a prominent European specialty pharmaceutical firm, has achieved a significant milestone with the approval of its groundbreaking medication, PEDMARQSI® (sodium thiosulfate anhydrous). Registered by the Australian Therapeutic Goods Administration (TGA), this drug aims to prevent hearing loss caused by cisplatin chemotherapy in pediatric patients aged 1 month to less than 18 years with localized, non-metastatic solid tumors.

The Need for PEDMARQSI®

Cisplatin is a crucial component of cancer therapy in children, particularly for treating solid tumors. However, a serious downside of this chemotherapy is its potential to cause permanent hearing loss, which can negatively affect a child's speech, language development, education, and overall quality of life. This condition poses a significant challenge, and there has been a notable demand for an effective preventive solution.

PEDMARQSI® emerges as the first and only registered treatment in Australia specifically designed to address this issue. By mitigating the risks of cisplatin-induced ototoxicity, PEDMARQSI® provides a vital option for families and healthcare professionals dedicated to improving pediatric oncology outcomes.

Insights from Norgine's Leadership

Gus Rudolph, General Manager of Norgine Australia, expressed the importance of this approval, stating, "The TGA registration of PEDMARQSI® marks an important milestone for children, families, and healthcare professionals in Australia. Cisplatin-induced hearing loss can have lifelong consequences for young patients, and this approval provides an option specifically developed to help prevent this complication in eligible children receiving cisplatin chemotherapy."

This sentiment was echoed by Dr. David Gillen, Norgine’s Chief Medical Officer, who highlighted the severe implications that hearing loss can have on communication, learning, and overall quality of life in children undergoing cancer treatment. He emphasized the merit of having a preventative option now available, noting how this advancement marks a new era in supporting pediatric oncology care.

Clinical Trials and Regulatory Milestones

The TGA's decision to approve PEDMARQSI® was based on data derived from two pivotal Phase 3 clinical trials: SIOPEL 6 and COG ACCL0431. These trials demonstrated the drug’s potential safety and efficacy in preventing hearing loss in the targeted pediatric population.

Norgine's success in navigating regulatory pathways for such specialized therapies is indicative of its commitment to addressing unmet medical needs. In addition to gaining TGA registration in Australia, PEDMARQSI® previously achieved Paediatric Use Marketing Authorisation (PUMA) from the European Medicines Agency in May 2023, along with approvals in the UK and Switzerland earlier this year.

Looking Ahead

While PEDMARQSI® is not currently listed on the Pharmaceutical Benefits Scheme (PBS), Norgine is focused on collaborating with healthcare stakeholders to ensure access for eligible patients in Australia. The company's mission is to deliver innovative medicines that truly change lives, driven by the conviction that every scientific breakthrough should reach those in need.

About Norgine

Norgine is a mid-sized European pharmaceutical company that employs approximately 1,500 individuals and generates around $650 million in annual sales. With an innovative approach to developing, commercializing, and manufacturing medications, Norgine is committed to tackling significant health challenges, ranging from common ailments to rare diseases like childhood cancer. The company strives to expand the availability of transformative medicines across Europe, Australia, and New Zealand.

Conclusion

The approval of PEDMARQSI® by the TGA represents a crucial step towards better treatment options for pediatric cancer patients in Australia. It underscores Norgine's dedication to innovation and its mission to address the pressing medical needs of vulnerable populations. As the healthcare community rallies to support children undergoing cisplatin therapy, PEDMARQSI® stands as a beacon of hope to improve the quality of life for young cancer patients and their families.