Fennec Pharmaceuticals Inc (NASDAQ:FENC) (TSX:FRX), a Durham, NC-based pharmaceutical company focused on developing therapies for the prevention of platinum-induced ototoxicity in pediatric patients, announced that the US Food and Drug Administration (FDA) has granted PEDMARK™(a unique formulation of sodium thiosulfate) Breakthrough Therapy designation for prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB).
“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 CEO of Fennec. “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.”
According to the FDA, Breakthrough Therapy designation is given when preliminary clinical evidence has been provided to show that a treatment effect may represent substantial improvement over available therapies for the treatment of a serious condition. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Additional information is available under the FDA guidance for Industry Expedited Programs for Serious Conditions—Drugs and Biologics: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf
About PEDMARK™ (Sodium Thiosulfate (STS))
Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity in some patients, and can be particularly harmful to the survivors of pediatric cancer.
According to Fennec, STS 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 are 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.
Source: Fennec Pharmaceuticals
Image: Fennec Pharmaceuticals