Pedmarqsi® (anhydrous sodium thiosulfate) recommended by NICE for patients 1 month to 17 years of age in England and Wales for the prevention of cisplatin-induced hearing loss

• Pedmarqsi® (anhydrous sodium thiosulfate) becomes the first and only treatment to be made available within NHS England and Wales for the prevention of cisplatin-induced ototoxicity (hearing loss) in children and young people (1 month-17 years of age).
• The data from 2 open-label, randomised Phase 3 trials, SIOPEL 6 (pivotal)¹ and the Clinical Oncology Group [COG] Protocol ACCL0431², showed that there was around a 50% reduction in the occurrence of cisplatin-induced ototoxicity in patients treated with cisplatin and sodium thiosulfate vs. those treated with cisplatin alone.
• There is a clear unmet need for the prevention of hearing loss caused by cisplatin as until now, there have been no preventative pharmacological interventions available, despite the significant lifelong impact hearing loss has on patients.

20th December, 2024, Harefield UK, – Norgine Pharmaceuticals Ltd, a leading European specialty pharmaceutical company, announced today the positive final draft guidance (FDG) from the National Institute for Health and Care Excellence (NICE) recommending Pedmarqsi® (anhydrous sodium thiosulfate) for the prevention of cisplatin-induced ototoxicity (CIO) in patients (aged 1 month to 17 years) with localised, non-metastatic, solid tumours.

Cisplatin is an effective chemotherapy agent for the treatment of childhood cancers, but it can cause irreversible, bilateral hearing loss – ototoxicity – in around 60% of children. This can occur from the first dose of cisplatin, progressing after each subsequent dose. Hearing loss can have a profound, multifaceted impact on a child's development, affecting speech, language acquisition, psycho-social and cognitive development, and independence, leading to significant, long-term detrimental effects on their quality-of-life.

Pedmarqsi® is the first and only treatment specifically tailored for use in paediatric patients with localised solid tumours for the prevention of ototoxicity. This decision from NICE represents an important milestone for the young patients who will benefit from this medicine.

Mark Ashton, UK General Manager, Norgine said: “We are delighted that the National Institute for Health and Care Excellence (NICE) has recommended Pedmarqsi®, highlighting the value it brings to this important unmet medical need. It also recognises the positive impact that this treatment could have not only on the quality of life, but importantly, on the future of these children. At Norgine, we are excited to build our presence in paediatric oncology, as we aim to bring transformative and life-changing medicines to children in the UK.”