Pedmarqsi
Authorised
This medicine is authorised for use in the European Union
sodium thiosulfate
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
Pedmarqsi is a medicine used in children aged 1 month to less than 18 years old to reduce the risk of hearing loss caused by the cancer medicine cisplatin when used to treat solid tumours that have not spread.
Pedmarqsi contains the active substance sodium thiosulfate.
Pedmarqsi can only be obtained with a prescription and must be given in a hospital under the supervision of an appropriately qualified doctor. It is given as an infusion (drip) into a vein lasting 15 minutes, exactly 6 hours after the patient has received cisplatin.
For more information about using this medicine, see the package leaflet or contact you or your child’s doctor or pharmacist.
The way Pedmarqsi works is not fully understood, but the active substance, sodium thiosulfate, is thought to act by binding to and blocking the action of cisplatin that has not been taken up by cells and preventing damage to cells caused by molecules known as ‘oxygen free radicals’. These combined actions are expected to help protect the ear against hearing loss caused by cisplatin.
Two studies found that Pedmarqsi reduced the risk of hearing loss in children aged 1 month to 18 years who were receiving cisplatin to treat solid tumours.
The first study involved 114 children with hepatoblastoma (a cancer of the liver), with an average age of about 19 months. The results showed that 35% (20 out of 57) of children who received Pedmarqsi 6 hours after each dose of cisplatin developed hearing loss compared with 67% (35 out of 52) of children who only received cisplatin.
The second study involved 125 children aged 1 month to 18 years with different types of cancer, including hepatoblastoma, neuroblastoma (a cancer of immature nerve cells) and tumours of the central nervous system. The study found that hearing loss was experienced by 29% (14 out of 49) of children who received Pedmarqsi after each cisplatin dose compared with 56% (31 out of 55) of those who received only cisplatin.
For the full list of side effects and restrictions with Pedmarqsi, see the package leaflet.
The most common side effects with Pedmarqsi (which may affect more than 1 in 10 people) include vomiting, nausea (feeling sick), hypernatraemia (high blood levels of sodium), hypophosphataemia (low blood levels of phosphate) and hypokalaemia (low blood levels of potassium).
The most common serious side effects with Pedmarqsi (which may affect more than 1 in 10 people) include hypersensitivity (allergic reactions).
Pedmarqsi must not be used in infants under the age of 1 month.
Hearing loss due to cisplatin is an important clinical issue for which there were no treatment options available at the time of the authorisation of Pedmarqsi. Pedmarqsi has been shown to prevent hearing loss in children and adolescents caused by cisplatin treatment for certain cancers. In addition, the safety profile of Pedmarqsi is in line with that known for sodium thiosulfate when given for other uses and is considered acceptable. The European Medicines Agency therefore decided that Pedmarqsi’s benefits are greater than its risks and it can be authorised for use in the EU.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Pedmarqsi have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Pedmarqsi are continuously monitored. Suspected side effects reported with Pedmarqsi are carefully evaluated and any necessary action taken to protect patients.
Pedmarqsi received a marketing authorisation valid throughout the EU on 26 May 2023.
Further information on Pedmarqsi can be found on the Agency’s website: ema.europa.eu/medicines/human/EPAR/pedmarqsi.
Product information
Latest procedure affecting product information:
IAIN/0006/G
24/07/2024
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Pedmarqsi
- Active substance
- Sodium thiosulfate
- International non-proprietary name (INN) or common name
- sodium thiosulfate
- Therapeutic area (MeSH)
- Ear Diseases
- Ototoxicity
Pharmacotherapeutic group
All other therapeutic productsTherapeutic indication
Pedmarqsi is indicated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to < 18 years of age with localised, non-metastatic, solid tumours.
News on Pedmarqsi
This page was last updated on