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FDA Grants Breakthrough Therapy Designation to Fennec PEDMARK™

The US Food and Drug Administration (FDA) awarded breakthrough therapy designation to Fennec Pharmaceuticals’ PEDMARK™ (a unique formulation of sodium thiosulfate) for prevention of cisplatin-related ototoxicity in pediatric patients with standard-risk hepatoblastoma(SR-HB).

The Fast Track designation will facilitate development and accelerate the review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

It includes all of the fast track programme features, as well as more intensive FDA guidance on an efficient drug development programme.

In addition, the breakthrough therapy designation highlights the current lack of safe and effective treatments and overwhelming need to address this serious condition.

PEDMARK™ (Sodium Thiosulfate (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.

SIOPEL 6 is a multi-centre open-label randomised phase 3 study evaluating the efficacy of STS in reducing ototoxicity in patients receiving cisplatin monotherapy for standard risk hepatoblastoma.  

In the study, the COG ACCL0431 protocol enrolled one of five childhood cancers typically treated with thorough cisplatin therapy for localised and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma.  

SIOPEL 6 enrolled only hepatoblastoma patients with localised tumors. COG ACCL0431 final results were published in the Lancet Oncology.

The study confirmed that the addition of STS reduces the incidence of cisplatin-induced hearing loss without any evidence of tumour protection.

Among the 99 evaluable patients, hearing loss occurred in 30/45=67Percent treated with Cisplatin (Cis) alone and in 20/54=37.0Percent treated with Cis+STS, corresponding to a relative risk of 0.56(P=0.0033).

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