Pediatric Hepatoblastoma Treatment Gets Fast Tracked by FDA

The U.S. Food and Drug Administration (FDA) has granted Pedmark, a unique combination of cisplatin and sodium thiosulfate (STS), Fast Track designation for the prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB).

If approved, the regimen would be the first available therapy for this condition.

Two recent Phase 3 clinical trials of survival and reduction of ototoxicity with STS — ACCL0431 and SIOPEL 6 – have been completed. The first was intended to compare the efficacy of STS against observation in preventing hearing loss in young patients receiving cisplatin for the treatment of newly diagnosed hepatoblastoma, as well as five other malignancies. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. Both met their primary endpoints.

"We believe the receipt of Fast Track designation from the FDA highlights the serious nature of hearing loss that patients have following cisplatin chemotherapy and the current lack of safe and effective treatments," said Rosty Raykov, President and Chief Executive Officer of Fennec in a press release. "We look forward to the more frequent interactions with the Agency that the Fast Track designation provides, as we prepare for the NDA filing.”

ACCL0431 enrolled 131 participants, each of whom received STS IV (16 g/m2 or 533 mg per kg for patients whose therapeutic protocol administers cisplatin on a per kg basis due to young age or small body) over 15 minutes beginning 6 hours after the completion of each cisplatin infusion.

SIOPEL 6 was started in October 2007 and completed enrollment at the end of 2014. Initiated in the United Kingdom, eventually 52 sites from 11 countries enrolled a total of 109 evaluable patients. The patients were randomized and the cisplatin and STS combination was generally well-tolerated.

Standard of care for the condition has long been platinum-based therapies, however, Unfortunately, they frequently cause ototoxicity in many patients, and are particularly harmful to survivors of pediatric cancer. Fennec is focused on the development of STS for the prevention of platinum-induced ototoxicity in this indication, and STS has previously received Orphan Drug Designation in the U.S.

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