Norgine has received Australian Therapeutic Goods Administration registration for PEDMARQSI (sodium thiosulfate anhydrous) for preventing cisplatin-induced hearing loss in children aged one month to under 18 years with localised, non-metastatic solid tumours. PEDMARQSI is the first and only treatment registered in Australia for preventing cisplatin-induced ototoxicity in paediatric patients. Cisplatin chemotherapy carries substantial risk of permanent hearing loss, which can affect speech development, education and quality of life in children. The TGA registration is based on data from two Phase 3 clinical trials. The approval follows previous authorisations in Europe, the United Kingdom and Switzerland. PEDMARQSI is not currently included on Australia's Pharmaceutical Benefits Scheme. Norgine is a European specialty pharmaceutical company with 1,500 employees generating approximately $650 million in annual sales.

Norgine has received European Commission marketing authorisation for XOLREMDI (mavorixafor), the first approved treatment for WHIM syndrome in the European Union. The approval, granted under exceptional circumstances, follows a positive opinion from the EMA's Committee for Medicinal Products for Human Use. XOLREMDI is indicated for patients aged 12 and older with WHIM syndrome, an ultra-rare primary immunodeficiency that impairs white blood cell release from bone marrow, increasing infection risk. The approval is supported by results from a pivotal Phase 3 trial involving 31 patients. Norgine will commercialise mavorixafor across Europe, Australia and New Zealand under a licensing agreement with X4 Pharmaceuticals announced in January 2025. The company will handle market access and commercialisation, whilst X4 will manufacture and supply the treatment.