RESEARCH TRIANGLE PARK, NORTH CAROLINA — Fennec Pharmaceuticals Inc., a specialty pharmaceutical company, announced that the U.S. 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. 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.”
–Rosty Raykov, CEO of Fennec Pharmaceuticals, Inc.
PEDMARK has been granted both Fast Track Designation and Breakthrough Therapy Designation by the FDA. 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.
Cisplatin and other platinum compounds are essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.
In the U.S. and Europe, it is estimated that, annually, over 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.
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.
About Fennec Pharmaceuticals
Fennec Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the development of PEDMARK™ for the prevention of platinum-induced ototoxicity in pediatric patients. Further, PEDMARK has received Orphan Drug Designation in the U.S. for this potential use. Fennec has a license agreement with Oregon Health and Science University (OHSU) for exclusive worldwide license rights to intellectual property directed to sodium thiosulfate and its use for chemoprotection, including the prevention of ototoxicity induced by platinum chemotherapy, in humans. For more information, please visit www.fennecpharma.com