A “groundbreaking” drug which helps to prevent hearing loss among children undergoing cancer treatment has been given the green light for NHS use.
Chemotherapy drug cisplatin is widely used to treat many cancers, especially in children.
But experts have said that around 60% of children treated with cisplatin experience permanent hearing loss as the drug builds up in the cochlea in the inner ear and damages the hair cells, which are unable to grow back.
Some 283 new cases of “ototoxic hearing loss” were diagnosed in people under 18 in England between 2022 and 2023.
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.
Now the National Institute for Health and Care Excellence (Nice) has recommended a first of its kind treatment which has been shown to reduce the risk of hearing loss among babies, children and young people who are being treated with cisplatin.
Nice estimates that around 60 children and young people in England will benefit from Pedmarqsi in the first year of its rollout.
Draft guidance from Nice recommends Pedmarqsi, also known as anhydrous sodium thiosulfate – which is made by Norgine, for children undergoing this form of chemotherapy aged one month to 17 years old in final draft guidance.
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.
The treatment is given via an IV drip and prevents damage to cells in the ear caused by cisplatin.
It does not reduce the effectiveness of the chemotherapy.
Nice said evidence from clinical trials shows that the treatment almost halves the rate of hearing loss in children receiving cisplatin chemotherapy.
It added that if children who take the treatment do still develop hearing loss, the clinical trial evidence shows it is less severe.
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.”
Ralph Holme, director of research at the hearing loss charity RNID, said; “We are thrilled by the announcement that this groundbreaking drug will be made available to young patients undergoing cancer treatment to prevent hearing loss as a side effect of their cisplatin chemotherapy.
“We look forward to seeing it being rolled out in hospitals across the country, and we are hopeful that all children who may benefit will soon have access to this vital treatment.
“It is the first drug specifically developed to prevent hearing loss to be recommended for use in the NHS. This is an important milestone that will give confidence to those investing in and developing treatments for hearing loss that they can by successfully brought to market.”
Mark Ashton, UK general manager at Norgine, said: “We are delighted that the National Institute for Health and Care Excellence 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.”
