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 and granted Priority Review for the company’s New Drug Application (NDA) for PEDMARK™ (a unique formulation of sodium thiosulfate).
PEDMARK is an investigational drug 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 FDA filing acceptance of our NDA and granting of Priority Review represents a significant milestone in the development of PEDMARK and we look forward to working closely with the Agency during this review process”
–Rosty Raykov, CEO of Fennec
The FDA grants Priority Review to applications for medicines that treat a serious condition, and, if approved, would demonstrate the potential to be a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition. Priority Review designation shortens the review period from the standard ten months to six months from the submission of the NDA. The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of August 10, 2020 for the completion of FDA’s review.
Preventing Hearing Loss from Chemotherapy
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 cooperative 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 one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.
The FDA has accepted for filing the Company’s New Drug Application (NDA) for PEDMARK™ and has granted Priority Review. The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename to be determined) is currently under evaluation by the European Medicines Agency (EMA). PEDMARK has received Breakthrough Therapy and Fast Track Designation by 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 STS and its use for chemoprotection, including the prevention of ototoxicity induced by platinum chemotherapy, in humans.