NICE approves new drug ‘Pedmarqsi’ to prevent hearing loss in children with cancer

Evidence from clinical trials showed the drug almost halves the rate of hearing loss in children receiving cisplatin chemotherapy

Children undergoing cancer treatment in England will soon have access to a groundbreaking new drug designed to prevent hearing loss caused by cisplatin chemotherapy.

National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending anhydrous sodium thiosulfate, marketed as Pedmarqsi by Norgine, for children aged one month to 17 years with solid tumours that have not spread to other parts of the body.

Cisplatin, a widely used chemotherapy medicine for treating childhood cancers, is highly effective but often leads to a side effect known as ototoxicity—damage to the inner ear caused by the drug's buildup, resulting in irreversible hearing loss.

Statistics show that approximately 60 per cent of children undergoing cisplatin-based treatments develop hearing loss, with 283 new cases diagnosed among individuals under 18 in England between 2022 and 2023.

Pedmarqsi is expected to be available within three months of NICE’s final guidance publication, and it is estimated to benefit about 60 children and young people in its first year of use.

The drug will be available to the NHS at a discounted rate through a confidential commercial arrangement with the company.

Helen Knight, director of medicines evaluation at NICE, said: “Hearing loss due to cancer treatment is devastating for children and their families so we are pleased to be able to recommend this ground-breaking treatment.

“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.”

How the drug works

Administered as an infusion by healthcare professionals, anhydrous sodium thiosulfate works by binding to and blocking the action of cisplatin that hasn’t been absorbed by cells, preventing it from damaging inner ear cells.

Importantly, the drug does not impact the effectiveness of cisplatin chemotherapy.

Evidence from two clinical trials showed the treatment almost halves the rate of hearing loss in children receiving cisplatin chemotherapy.

One trial found that hearing loss occurred in only 32.7 per cent of children treated with both cisplatin and anhydrous sodium thiosulfate, compared to 63 per cent of those who received cisplatin alone.

Another trial reported hearing loss in 28.6 per cent of children given the new treatment, compared to 56.4 per cent of those treated with cisplatin only.

Additionally, any hearing loss in children treated with the drug was generally less severe.