Inozyme Pharma Inc 2024年度报告

The Rare Pediatric Disease Priority Review Voucher program was not reauthorized by Congress in 2024 and expired onDecember 31, 2024. Thus, under the current statutory sunset provisions, FDA may only award PRVs for approved rare pediatricdisease product applications if sponsors had rare pediatric disease designation for the drug or biologic granted by September 30, 2024.The FDA may not award any rare pediatric disease PRVs after September 30, 2026. In September 2020, we received rare pediatricdisease designation from the FDA for INZ-701 for the treatment of ENPP1 Deficiency. However, the FDA may determine that a BLAfor INZ-701 or one or more of our other product candidates does not meet the eligibility criteria for a PRV upon approval. Moreover,if we do not obtain approval of our BLA for INZ-701 by September 30, 2026, and if the program is not further extended byCongressional action, we will not receive a PRV. The FDA, EMA, or other comparable foreign regulatory authorities could require the clearance or approval of a companiondiagnostic device as a condition of approval for any product candidate that requires or would commercially benefit from such tests.Failure to successfully validate, develop and obtain regulatory clearance or approval for companion diagnostics on a timely basisor at all could harm our product development strategy and we may not realize the commercial potential of any such productcandidate. If safe and effective use of any of our other product candidates depends on an in vitro diagnostic, then the FDA generallywill require approval or clearance of that diagnostic, known as a companion diagnostic, at the same time that the FDA approves ourproduct candidates. The process of obtaining or creating such diagnostic is time consuming and costly. Companion diagnostics, whichprovide information that is essential for the safe and effective use of a corresponding therapeutic product, are subject to regulation bythe FDA, EMA, and other comparable foreign regulatory authorities as medical devices and require separate regulatory approval fromtherapeutic approval prior to commercialization. The FDA previously has required in vitro companion diagnostics intended to selectthe patients who will respond to a product candidate to obtain pre-market approval ("PMA") simultaneously with approval of thetherapeutic candidate. The PMA process, including the gathering of preclinical and clinical data and the submission and review by theFDA, can take several years or longer. It involves a rigorous pre-market review during which the sponsor must prepare and provideFDA with reasonable assurance of the device’s safety and effectiveness and information about the device and its componentsregarding, among other things, device design, manufacturing, and labeling. After a device is placed on the market, it remains subjectto significant regulatory requirements, including requirements governing development, testing, manufacturing, distribution, marketing,promotion, labeling, import, export, record-keeping, and adverse event reporting. Given our limited experience in developing and commercializing diagnostics, we do not plan to develop companiondiagnostics internally and thus will be dependent on the sustained cooperation and effort of third-party collaborators in developing andobtaining approval for these companion diagnostics. We may not be able to enter into arrangements with a provider to develop acompanion diagnostic for use in connection with a registrational trial for our product candidates or for commercialization of ourproduct candidates, or do so on commercially reasonable terms, which could adversely affect and/or delay the development orcommercialization of our product candidates. We and our future collaborators may encounter difficulties in developing and obtainingapproval for the companion diagnostics, including issues relating to selectivity/specificity, analytical validation, reproducibility, orclinical validation. Any delay or failure by our collaborators to develop or obtain regulatory approval of the companion diagnosticscould delay or prevent approval of our product candidates. In addition, we, our collaborators or third parties may encounter productiondifficulties that could constrain the supply of the companion diagnostics, and both they and we may have difficulties gainingacceptance of the use of the companion diagnostics by physicians. We believe that adoption of screening and treatment into clinical practice guidelines is important for payer access,reimbursement, utilization in medical practice and commercial success, but both our collaborators and we may have difficulty gainingacceptance of the companion diagnostic into clinical practice guidelines. If such companion diagnostics fail to gain market acceptance,it would have an adverse effect on our ability to derive revenues from sales, if any, of any of our product candidates that are approvedfor commercial sale. In addition, any companion diagno