Australia’s Therapeutic Goods Administration (TGA) is evaluating Norgine Pharmaceutical’s PEDMARQSI for the prevention of hearing loss induced by cisplatin chemotherapy in children.
The proposed new indication is “for the prevention of damage to parts of the ear that is sometimes caused by cisplatin chemotherapy”.
Trials show the treatment halves the incidence of ototoxicity, or permanent hearing loss, in those who receive it while undergoing cisplatin chemotherapy compared with those who just receive the chemotherapy.
“PEDMARQSI is to be used in children aged one month to under 18 years with localised solid tumours that have not spread to other parts of the body,” an announcement on the prescription medicines evaluation section of the TGA website said.
The treatment is being used in the US, Germany and the UK.
The TGA accepted the application in January 2025 from the drug’s sponsor Norgine Pty Ltd. Its active ingredient is sodium thiosulfate anhydrous.
Approved in Germany
Meanwhile Fennec Pharmaceuticals, a company focused on development and commercialisation of the treatment, announced its commercial launch in Germany in February 2025 and said this would accelerate the global commercialisation strategy.
It said the treatment was the first and only approved therapy in the EU and United Kingdom for the prevention of ototoxicity, or hearing loss induced by cisplatin chemotherapy, in patients aged from one month to under 18 years of age with localised, non-metastatic solid tumours.
“In March 2024, Fennec entered into an exclusive licensing agreement under which Norgine will commercialise PEDMARQSI® in Europe, Australia and New Zealand,” a Fennec media release said.
“Under the terms of the previously announced exclusive licensing agreement with Norgine, Fennec received approximately $43 million in an upfront payment and will receive up to approximately $230 million in additional commercial and regulatory milestone payments along with double-digit tiered royalties on net sales of PEDMARQSI starting in the mid-teens and growing to the mid-twenties.”
A 50% reduction in ototoxicity
Data from two open-label, randomised phase three trials, SIOPEL 6 (pivotal) and the Clinical Oncology Group (COG) Protocol ACCL0431, demonstrated an approximate 50% reduction in the occurrence of cisplatin-induced ototoxicity in patients treated with cisplatin and sodium thiosulfate versus those treated with cisplatin alone.
The European Commission granted PEDMARQSI marketing authorisation in June 2023 under the Paediatric Use Marketing Authorization (PUMA), which includes eight years plus two years of data and market protection.
“Cisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many paediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of paediatric cancer,” Fennec said.
It said the incidence of ototoxicity depended on dose and duration of chemotherapy, and many of the children required lifelong hearing aids or cochlear implants.
“Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational achievement,” the media release stated.
Fennec said that in the US and Europe, it was estimated that more than 10,000 children may receive platinum-based chemotherapy annually.
In America, the treatment is branded PEDMARK sodium thiosulfate injection for intravenous use. The FDA approved it in September 2022 as the first and only approved therapy to reduce risk of ototoxicity associated with cisplatin treatment in paediatric patients with localised, non-metastatic, solid tumors. It is also recommended there for adolescents and young adults.
PEDMARK received European Commission approval in June 2023 and UK approval in October 2023 under the name PEDMARQSI.
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