The resubmission for PEDMARK follows receipt of final minutes from a Type A meeting with the FDA. Importantly, the Complete Response Letter (CRL) received on
'We are pleased to have resubmitted the NDA for PEDMARKTM and look forward to working with the FDA through the review process,' said
About PEDMARK
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 cancer.
In the
PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.
The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename PEDMARQSI) is currently under evaluation by the
About
Forward Looking Statements
Except for historical information described in this press release, all other statements are forward-looking. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'estimate,' 'intend,' 'may,' 'will,' or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include the Company's expectations regarding its interactions and communications with the FDA, including the Company's expectations and goals respecting the resolution the issues raised in the CRL and the Company's plans to address them, and the anticipated timing of the Company's finalization and filing of an NDA resubmission for PEDMARK. Forward-looking statements are subject to certain risks and uncertainties inherent in the Company's business that could cause actual results to vary, including such risks and uncertainties relating to the Company's reliance on third party manufacturing, the risk that unforeseen factors may delay the resubmission of the NDA, the risks of delays in or failure to obtain FDA approval of PEDMARK, the risks relating to the Company's and its manufacturer's ability to adequately address the concerns identified in the CRL, the risk that the resubmission of the NDA to the FDA will not be satisfactory, that regulatory and guideline developments may change, scientific data and/or manufacturing capabilities may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results may not be replicated in actual patient settings, unforeseen global instability, including political instability, or instability from an outbreak of pandemic or contagious disease, such as the novel coronavirus (COVID-19), or surrounding the duration and severity of an outbreak, protection offered by the Company's patents and patent applications may be challenged, invalidated or circumvented by its competitors, the available market for the Company's products will not be as large as expected, the Company's products will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical studies, the Company may not meet its future capital requirements in different countries and municipalities, and other risks detailed from time to time in the Company's filings with the
For a more detailed discussion of related risk factors, please refer to our public filings available at www.sec.gov and www.sedar.com.
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