The FDA has approved sodium thiosulfate (Pedmark) as the first treatment for cisplatin-related ototoxicity in pediatric patients, Fennec Pharmaceuticals announced.
The approved indication is to reduce the risk of ototoxicity associated with cisplatin in pediatric patients ages 1 month or older with solid non-metastatic tumors.
“Historically, there have been no approved treatments for preventing cisplatin-induced hearing loss,” said David R. Freyer, DO, of Children’s Hospital Los Angeles, in a statement from the company. “As a physician focused in pediatric cancer for many years, and a primary investigator in the pivotal Pedmark phase III clinical oncology group trial, the FDA approval addresses an enormous unmet need, and for many children and young adults, has the potential to greatly improve everyday life for patients.”
Support for the approval came from two phase III open-label, randomized trials that compared cisplatin-based chemotherapy plus sodium thiosulfate and cisplatin-based chemotherapy alone in pediatric patients. In both studies, the incidence of ototoxicity with hearing loss was reduced by 50% or more among patients who received sodium thiosulfate. In both trials combined, the most common adverse events in the sodium thiosulfate-treated patients with ≥5% difference versus the control arm were vomiting, infection, nausea, decreased hemoglobin, hypernatremia, and hypokalemia.
Despite recent advances in treatment for pediatric malignancies, cisplatin-based chemotherapy remains the treatment of choice in many cases. Though effective, cisplatin confers a well-documented risk of ototoxicity, and causes irreversible bilateral hearing loss in about 60% of children treated with the drug. Interventions such as cochlear implants and hearing aids are beneficial only after hearing loss has occurred.
“Hearing loss can have a profound impact on a person’s life, especially in children who are critically dependent upon normal hearing for cognitive, psychosocial, and speech development,” said Penelope R. Brock, MD, PhD, of Great Ormond Street Hospital in London, in a statement from Fennec. “Incorporating Pedmark into current treatment strategies with the goal to preserve hearing in children and young adults without reducing the effectiveness of their cisplatin treatment – is a welcome step towards helping to improve long-term outcomes for these patients.”
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