A new drug that is proven to lower the risk of hearing loss in children with cancer will be presented at the Hearing Therapeutics Summit, organised by RNID, with the UCL Ear Institute and UCLH NIHR Biomedical Research Centre.
Trials for the drug, called PEDMARK©, showed the risk of hearing loss in children undergoing cisplatin-based cancer treatment was reduced by up to 42%. Children taking part in the trial were aged one month and older and received cisplatin-based cancer treatment for solid tumours that had not spread to other parts of the body.
Cisplatin is a powerful chemotherapy medicine used to treat many cancers in children. Over time, cisplatin builds up in the cochlea in the inner ear and damages the hair cells which are unable to grow back. More than 70% of children treated with cisplatin experience permanent hearing loss.
PEDMARK© was granted approval by the U.S. Food and Drug Administration in September 2022. In June 2023, Fennec Pharmaceuticals was granted marketing authorisation for Pedmarqsi™ – known as PEDMARK© in the U.S. – by the European Commission.
Pedmarqsi™ is the first and only approved therapy in the EU for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients aged between one month and 18 years with localised, non-metastatic, solid tumors.
Fennec is evaluating the best commercial pathway in Europe and for the rest of the world and looks forward to making Pedmarqsi™ available in the UK as soon as possible.
Dr Ananya Bhattacharya, Head of Medical Affairs & Safety at Fennec Pharmaceutical, said:
“The recent approval of Pedmarqsi™ brings the approximately four thousand children in Europe with cancer who are treated with cisplatin every year one step closer to the first approved treatment option to prevent the devastating consequences of hearing loss following the use of cisplatin chemotherapy, an indispensable treatment of choice in many pediatric cancer cases. Fennec is very pleased that Pedmarqsi™ offers patients and their families the opportunity to avoid this life impacting side effect and can give children the opportunity to live healthy, happy, and fully integrated lives after overcoming cancer.”
Dr Ralph Holme, Director of Research and Insight at RNID, said:
“RNID and the hearing research community is incredibly excited by the recent approval of Pedmarqsi™, and we look forward to when this important drug will be made available in the UK to children undergoing cancer treatment.
“The majority of children treated with cisplatin chemotherapy currently experience permanent hearing loss, but this drug could turn the tables so that more children are able to enjoy their hearing for years to come.
“At RNID we want there to be a range of treatments to prevent hearing loss and restore hearing for those who need and want them, and our Hearing Therapeutics Summit is an important way to bring the research community together in order to share insight and speed up the development of new treatments.”
The Hearing Therapeutics Summit will take place on Saturday 2 September with experts, researchers and industry coming together to discuss bringing new treatments to patients, challenges in hearing therapeutics development and bridging the gap between academia and industry.