Pedmarqsi® (sodium thiosulfate solution for infusion) becomes the first and only treatment to be made available within NHS Scotland for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumours.1
The data from 2 open-label, randomised Phase 3 trials, SIOPEL 6 (pivotal)2 and the Clinical Oncology Group [COG] ACCL04313, 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.
12th May, 2025, Uxbridge UK – Norgine Pharmaceuticals Ltd, a leading European specialty pharmaceutical company, announced today that, following a full submission assessed under the orphan equivalent medicine process, the Scottish Medicines Consortium (SMC) has accepted Pedmarqsi® for use in Scotland for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumours. This decision follows that of the National Institute for Health and Care Excellence (NICE) on the 22nd of January 2025, when Pedmarqsi® was recommended within England and Wales4.
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, education, psycho-social and cognitive development, and independence, resulting in a significant, lifelong detrimental effect on patients’ quality-of-life.
Pedmarqsi® is the first and only treatment for the prevention of ototoxicity which is specifically tailored for use in paediatric patients with localised, non-metastatic, solid tumours. This decision from the SMC represents an important milestone for the young patients who will benefit from this medicine.
Mark Ashton, UK General Manager, Norgine said: “Following the recommendation of NICE in January of this year, we are delighted that the SMC has accepted Pedmarqsi® for use in Scotland, 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.”
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Media Contacts
Annabel Cowper, Norgine Pharmaceuticals Limited (ACowper1@norgine.com)
NOTES TO EDITORS
About PEDMARQSI®
Pedmarqsi® is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss in patients 1 month to <18 years of age. It is the first and only preventative treatment developed for cisplatin-induced ototoxicity to support children and adolescent patients with localised, non-metastatic solid tumour cancers.5
Pedmarqsi® was granted marketing authorisation by the EMA in May 2023 (under the paediatric-use marketing authorisation (PUMA) programme) and the MHRA in October 2023. Marketing authorisations were based on safety and efficacy data from two open-label, randomised Phase III trials: SIOPEL 6 (pivotal)2 and Clinical Oncology Group [COG] ACCL0431.3
The SIOPEL 6 trial demonstrated that sodium thiosulfate significantly reduced the occurrence of cisplatin-induced ototoxicity by 48% (relative risk 0.52 (0.349, 0.778), p<0.001; 30% absolute risk reduction) in patients treated with cisplatin + sodium thiosulfate vs those in the cisplatin control group, with the efficacy of chemotherapy being maintained in the cisplatin + sodium thiosulfate group. Event-free survival (EFS) (82% vs 79%) and overall survival (OS) (98% vs 92%) rates were similar between the cisplatin + sodium thiosulfate vs the cisplatin control group respectively2.
Similarly, in COG ACCL0431, the likelihood of hearing loss was significantly lower by 48% in the cisplatin + sodium thiosulfate group vs the cisplatin control group (relative risk 0.516 (0.318, 0.839), p = 0.0040; 27.8% absolute risk reduction). In the total population, EFS (54% vs 64%) and OS (70% vs 87%) rates were similar between the cisplatin + sodium thiosulfate vs the cisplatin control group respectively. In a post-hoc analysis of patients with localised disease only, there were also nonsignificant differences in the EFS (60% vs 66%) and OS (83% vs 89%) rates between the treatment groups.3
Trials showed a favourable tolerability profile with nausea and vomiting being the most common adverse effects with Pedmarqsi® 1, and infection, anaemia and neutropenia being the most common grade 3-4 adverse events.2
About Norgine Ltd
Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people’s lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access.
Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021.
Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world.
NORGINE and the sail logo are trademarks of the Norgine group of companies.
References
Brock PR, et al. N Engl J Med 2018; 378(25): 2376–2385
Freyer DR, et al. Lancet Oncol 2017; 18(1): 63–74
https://www.nice.org.uk/guidance/ta1034 (accessed April 2025)
Norgine websites
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