Fennec Pharmaceuticals Inc (NASDAQ: FENC; TSX: FRX), a specialty pharmaceutical company, announced that the US Food and Drug Administration (FDA) has accepted for filing the resubmission of its New Drug Application (NDA) for PEDMARK (a unique formulation of sodium thiosulfate) for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to < 18 years of age with localized, non-metastatic, solid tumors. The Prescription Drug User Fee Act (PDUFA) target action date has been set for November 27, 2021.
“We are pleased that the FDA has accepted our PEDMARK resubmission,” said Rosty Raykov, CEO of Fennec Pharmaceuticals, Inc. “We look forward to working closely with the FDA through the review process. We are committed to bringing this treatment to children receiving cisplatin chemotherapy, an area of high unmet medical need. If approved, PEDMARK stands to be the first FDA-approved therapy to reduce the risk of cisplatin-induced ototoxicity in pediatric patients.”
PEDMARK has been granted both Fast Track Designation and Breakthrough Therapy Designation by the FDA, according to the company’s announcement. The Complete Response Letter (CRL) received on August 10, 2020, referred to deficiencies with the facility of the drug product manufacturer; no clinical safety or efficacy issues were identified and there was no requirement for further clinical data, according to Fennec.
Cisplatin and other platinum compounds are essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.
PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename PEDMARQSI) is currently under evaluation by the European Medicines Agency (EMA). PEDMARK received Breakthrough Therapy and Fast Track Designation from the FDA in March 2018.