RESEARCH TRIANGLE PARK, N.C.—Fennec Pharmaceuticals Inc. announced today that the company’s Marketing Authorization Application (MAA) for its investigational drug, a unique formulation of sodium thiosulfate (tradename to be determined in the EU) for infusion, has passed validation and is now under evaluation by the European Medicines Agency (EMA).
“The EMA validation of our marketing application represents another step forward in our efforts to address, if authorized, the critical unmet medical need to prevent or significantly reduce cisplatin induced hearing loss in children with localized solid tumors,” stated Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. “We look forward to working closely with the EMA during the review process with the goal of bringing this important product to patients.”
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 cancers. The MAA for Pediatric Use Marketing Authorization (PUMA), which was filed in early February this year, is for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to < 18 years of age with localized, non-metastatic solid tumors.
In the U.S. and Europe, it’s estimated that over 10,000 children may receive platinum-based chemotherapy annually. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss. Only cochlear implants have been shown to provide some benefit.
Back in August 2018, Fennec Pharmaceuticals noted that the Pediatric Committee (PDCO) of the EMA had accepted the company’s pediatric investigation plan (PIP) for the condition of the prevention of platinum-induced ototoxic hearing loss. The company was also advised in 2018 that sodium thiosulfate (unique formulation) is eligible for submission of a PUMA with incentives of automatic access to the centralized procedure, and up to 10 years of data and market protection. No deferred clinical studies were required in the positive opinion given by PDCO.
Earlier this February, Fennec Pharmaceuticals Inc. disclosed that it had completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Pedmark (Fennec’s formulation of sodium thiosulfate) for intravenous use. Fennec’s Pedmark regulatory submissions follow a pre-NDA meeting with the FDA in December 2018, after which Fennec initiated a rolling NDA.
Both applications are based upon clinical results from two pivotal Phase 3 clinical trials: SIOPEL 6, conducted by the International Childhood Liver Tumor Strategy Group (SIOPEL) with results published in the New England Journal of Medicine in June 2018; and ACCL0431, conducted by the Children’s Oncology Group (COG) with results published in Lancet Oncology in 2016. 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 completion of these regulatory submissions to the FDA and EMA are the culmination of many years of hard work, bringing us one step closer to achieving our mission. Fennec would like to thank the many parents, children and investigators who participated in the clinical trials, as well as our dedicated employees who helped us reach this important milestone,” noted Raykov in a press release. “We are well underway with commercialization readiness activities to support the potential launch of Pedmark and our transition to becoming a commercial-stage organization.”
The FDA has a 60-day review period to determine whether the Pedmark NDA is acceptable for filing. Pedmark has been granted Orphan Drug, Breakthrough Therapy, and Fast Track designations from the FDA. If Pedmark is granted a priority review, the Prescription Drug User Fee Act (PDUFA) action date is expected in the third quarter of 2020.