Fennec Pharma launches PEDMARK in US

PEDMARK was approved by the U.S. Food and Drug Administration (USFDA) in September 2022 based upon safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (Clinical Oncology Group [COG] Protocol ACCL0431 and SIOPEL 6), which compared PEDMARK plus cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients.

Advances in chemotherapy-based treatment approaches for pediatric patients with localized, solid tumors have improved, resulting in an 85 percent or higher five-year survival rate for these patients. However, use of platinum-based chemotherapy, still the treatment of choice in many cases, can be toxic to the ears and cisplatin treatment frequently causes permanent and irreversible bilateral (affecting both ears) hearing loss. Permanent hearing loss can be seen in approximately 60 percent of children treated with cisplatin and can be as high as 90 percent. Until now, interventions with management strategies such as cochlear implants and hearing aids only occurred after hearing loss had been detected and these interventions do not return normal hearing.

The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename PEDMARQSI) is currently under evaluation by the European Medicines Agency (EMA). Prior to its approval in the U.S., the FDA granted PEDMARK Priority Review designation in 2020 and Orphan Drug designation in 2004.

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