The Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to Pedmark™ (sodium thiosulfate; Fennec Pharmaceuticals) for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localized, non-metastatic, solid tumors.
The New Drug Application (NDA) application is supported by data from 2 pivotal open-label, randomized phase 3 trials (SIOPEL 6 and ACCL0431), which compared the efficacy of sodium thiosulfate plus cisplatin to cisplatin alone for the prevention of cisplatin-induced hearing loss. SIOPEL 6 enrolled 101 patients with hepatoblastoma. ACCL0431 included 105 patients with newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, or medulloblastoma.
Results from SIOPEL 6 showed that the incidence of hearing loss of grade 1 or greater was significantly less with sodium thiosulfate (33%; n=18/55) vs cisplatin alone (63%; n=29/46), indicating a 48% lower incidence of hearing loss in the sodium thiosulfate group (RR 0.52; 95% CI, 0.33-0.81; P=.002). Additionally, the 3-year rate of event-free survival was 82% for the cisplatin-sodium thiosulfate group and 79% for the cisplatin alone group; overall survival was 98% and 92%, respectively.
In the ACCL0431 trial, hearing loss was identified in 14 patients (28.6%) in the sodium thiosulfate arm compared with 31 patients (56.4%) in the control group (P=.00022). The likelihood of hearing loss was found to be significantly lower in the sodium thiosulfate group compared with the control group after adjusting for stratification variables (odds ratio 0.31; 95% CI, 0.13-0.73; P=.0036).
A Prescription Drug User Fee Act (PDUFA) target action date of August 10, 2020 has been assigned to this application. The FDA previously granted Breakthrough Therapy and Fast Track designations to Pedmark for this indication.
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