Pediatric Hepatoblastoma Therapy Sees More Regulatory Action


The U.S. FDA announced that Breakthrough Therapy Designation had been granted to Fennec Pharmaceuticals for its unique formulation of cisplatin and sodium thiosulfate (STS).

Yesterday, the U.S. Food and Drug Administration (FDA) announced that Breakthrough Therapy Designation had been granted to Fennec Pharmaceuticals for its unique formulation of cisplatin and sodium thiosulfate (STS).

The FDA granted the therapy, which will be marketed as Pedmark, Fast Track designation for the prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB) earlier this month. If approved, the regimen would be the first available therapy for this condition.

"The decision by the FDA to grant PEDMARK the first Breakthrough Therapy designation for the prevention of cisplatin ototoxicity reflects a recognition of the promising efficacy and safety data generated from SIOPEL 6 and COG ACCL0431 studies,” said Rosty Raykov, President and Chief Executive Officer of Fennec in a press release. We believe the recent receipt of Fast Track designation, and today, Breakthrough Therapy designation highlights the current lack of safe and effective treatments and overwhelming need to address this serious condition. This designation is another significant milestone for the advancement of PEDMARKTM, as we work closely with the Agency to expedite the NDA filing.”

In the recently completed Phase 3 SIOPEL 6 and COG ACCL0431 clinical trials, survival and reduction of ototoxicity with STS were observed. The former compared the efficacy of STS against observation in preventing hearing loss in young patients receiving cisplatin for the treatment of newly diagnosed hepatoblastoma and five additional malignancies, while the latter enrolled only hepatoblastoma patients with localized tumors. Both met primary endpoints.

The studies enrolled 131 and 109 patients, respectively. In ACCL0431, each 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 starting 6 hours after the completion of each cisplatin infusion. SIOPEL 6 was initiated in the United Kingdom and eventually conducted in 52 sites across 11 countries. The patients were randomized and the combination was generally well-tolerated.

Standard of care for SR-HB has long been platinum-based therapies, however, they commonly cause ototoxicity in many patients, and can be especially harmful to survivors of pediatric cancer. It is estimated that more than 10,000 children may receive platinum-based chemotherapy between the U.S. and Europe, and the incidence of hearing loss in these patients is dependent upon the dose and duration of the chemotherapy. As a result, many require lifelong hearing aids.

Fennec is focused on the development of STS for the prevention of platinum-induced ototoxicity in this indication.

STS has also previously received Orphan Drug Designation in the U.S.

For more from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter.

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