The National Institute for Health and Care Excellence (NICE) has issued a positive final draft guidance recommending Pedmarqsi (anhydrous sodium thiosulfate) for the prevention of cisplatin-induced hearing loss in children and young people aged 1 month to 17 years with localized, non-metastatic, solid tumors. This marks Pedmarqsi as the first and only treatment available within the National Health Service (NHS) in England and Wales for this condition.
Clinical Evidence and Efficacy
Two Phase 3 clinical trials, SIOPEL 6 and COG ACCL0431, demonstrated that Pedmarqsi reduces the occurrence of cisplatin-induced ototoxicity by approximately 50% compared to treatment with cisplatin alone. One trial showed a reduction from 63% hearing loss with cisplatin alone to 32.7% with the addition of anhydrous sodium thiosulfate. Another trial reported a decrease from 56.4% to 28.6% with the addition of Pedmarqsi. These results highlight the significant clinical benefit of Pedmarqsi in mitigating a severe side effect of cisplatin chemotherapy.
Addressing an Unmet Need
Cisplatin is a vital chemotherapeutic agent used to treat various pediatric malignancies. However, it is known to cause permanent and irreversible hearing loss (ototoxicity), which can severely impact the development of speech, language, and social-emotional skills in young patients. Prior to Pedmarqsi, there were no established preventative pharmacological interventions available to address this issue. The NICE recommendation fills a critical gap in supportive cancer care, offering a means to reduce long-term complications associated with cisplatin treatment.
Commercial and Financial Implications
Fennec Pharmaceuticals has an exclusive licensing agreement with Norgine Pharmaceuticals for the commercialization of Pedmarqsi in Europe, Australia, and New Zealand. The agreement includes an upfront payment of $43 million to Fennec, with potential additional payments of up to $230 million based on commercial and regulatory milestones. Furthermore, Fennec will receive double-digit tiered royalties on net sales of Pedmarqsi, starting in the mid-teens and growing to the mid-twenties.
Jeff Hackman, chief executive officer and director of Fennec Pharmaceuticals, stated, "Securing access to PEDMARQSI is a critical milestone for the cancer community in England and Wales to help reduce the risk of ototoxicity, or permanent hearing loss, associated with cisplatin treatment."
Impact on Patient Care
The availability of Pedmarqsi through the NHS is expected to significantly improve the quality of life for young cancer patients undergoing cisplatin chemotherapy. By reducing the risk of hearing loss, Pedmarqsi can help to preserve critical developmental milestones and improve long-term outcomes for these children. Helen Knight, Director of medicines evaluation at NICE, noted, "This is the first drug shown to prevent and reduce the impact of hearing loss, and it will have a life changing effect on the lives of children and young people."
It is estimated that just under 60 children and young people in England will be eligible for Pedmarqsi in the first year following recommendation of the drug.
