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
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– Norgine Pharmaceuticals Ltd, a leading European specialty pharmaceutical company, announced today the positive final draft guidance (FDG) from the National Institute for Health and Care Excellence (NICE) recommending Pedmarqsi® (anhydrous sodium thiosulfate) for the prevention of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.”
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Media Contacts
Charlotte Newman, Edelman Charlotte.Newman@edelman.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.
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)1 and Clinical Oncology Group [COG] Protocol ACCL04312.
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 respectively.
Similarly, in the COG Protocol 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 (64% vs 54%) and OS (87% vs 70%) 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 (66% vs 60%) and OS (89% vs 83%) rates between the treatment groups.
Trials showed a favourable tolerability profile with nausea and vomiting being the most common adverse effects with Pedmarqsi®, and infection, anaemia and neutropenia being the most common grade 3-4 adverse events3.
About Norgine Ltd
Norgine Ltd is a leading European specialty pharmaceutical and consumer healthcare company, with over €500 million of annual revenues and a 120-year history of delivering bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand.
Norgine has a significant heritage in the UK having international commercial operations in Harefield, Middlesex, and a manufacturing site in Hengoed, South Wales, where it employs over 600 people.
Our integrated approach – strong commercial capabilities, deep medical, regulatory, and clinical expertise, in-house manufacturing, robust supply networks, and best in class enabling functions – ensures that we can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually.
Today’s Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence, with the support of Goldman Sachs Alternatives and the founding Stein family. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into new therapeutic areas.
References
Job Number: UK-ONC-PED-2400012
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