Fennec Pharmaceuticals announced today that it expects to receive a Complete Response Letter (CRL) from the U.S. Food and Drug Administration for Pedmark (sodium thiosulfate), an investigational treatment for the prevention of ototoxicity associated with cisplatin chemotherapy in pediatric patients with localized, non-metastatic, solid tumors.
According to a recent press release from Fennec Pharmaceuticals, the FDA has indicated that, following a pre-approval inspection of the manufacturing facility found “deficiencies [had] been identified.”
Sodium thiosulfate was developed to prevent hearing loss seen with some common chemotherapies. More specifically, cisplatin and other platinum compounds are common and essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity, which is permanent, irreversible, and is particularly harmful to the survivors of pediatric cancer. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many children who receive platinum-based chemotherapies require lifelong hearing aids.
There is currently no established preventive agent for this type of hearing loss and only expensive, technically difficult, and sub-optimal cochlear implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development may have developmental delays in speech language while older children and adolescents may lack social-emotional development and have reduced educational achievement.
A unique formulation of sodium thiosulfate has been studied in two, phase 3 clinical trials (The Clinical Oncology Group Protocol [COP] ACCL0431 and SIOPEL 6). The COG ACCL0431 protocol enrolled pediatric patients with any one of the following: newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. These malignancies were chosen as they are typically treated with intensive cisplatin therapy. The SIOPEL 6 trial enrolled pediatric patients with localized tumors.
A Complete Response Letter means the FDA will no longer review the drug, in this case an orphan drug, for possible marketing approval. Instead, the developers of sodium thiosulfate, Fennec Pharmaceuticals, will have to submit a new application. Once the company officially receives the Letter, they plan to ask for a meeting with the FDA to discuss next steps to reapply.
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Reference
Fennec Pharmaceuticals [press release]. Fennec Pharmaceuticals Expects to Receive Complete Response Letter from the FDA for its New Drug Application for PEDMARK™ to Prevent Ototoxicity Associated with Cisplatin in Pediatric Patients with Localized, Non-Metastatic, Solid Tumors. Nov 29, 2021. https://www.globenewswire.com/news-release/2021/11/29/2341932/0/en/Fennec-Pharmaceuticals-Expects-to-Receive-Complete-Response-Letter-from-the-FDA-for-its-New-Drug-Application-for-PEDMARK-to-Prevent-Ototoxicity-Associated-with-Cisplatin-in-Pediatr.html
