Sangamo Therapeutics Receives U.S. FDA Fast Track Designation for Isaralgagene Civaparvovec for the Treatment of Fabry Disease
Fast Track designation aims to facilitate the development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Companies granted this designation are given the opportunity for more frequent interactions with the FDA. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met. The FDA has previously granted ST-920 Orphan Drug Designation.
“We are thrilled with the FDA’s decision to grant Fast Track Designation for ST-920. Fabry is a debilitating disease with life-long impact,” said
ST-920 is currently being evaluated in the Phase 1/2 STAAR study, with a total of 20 patients dosed to date. In
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate the safety and tolerability of isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. The STAAR study is enrolling patients who are on ERT, are ERT pseudo-naïve (defined as having been off ERT for six or more months), or who are ERT-naïve. The
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.
Sangamo Forward Looking Statements
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the ability of the Fast Track designation to facilitate the development and expedite the review of isaralgagene civaparvovec (ST-920), the ability to meet relevant criteria in order to be eligible to apply for Accelerated Approval and Priority Review, the therapeutic potential of isaralgagene civaparvovec, including its potential to improve the lives of patients without the need for ERT and to have a favorable effect on Fabry nephropathy, Sangamo’s expectation for, and timelines related to, the completion of dosing in the Phase 1/2 STAAR study, plans to meet with the FDA on the proposed Phase 3 study design, the planning for and the anticipated commencement of and dosing in a potential Phase 3 trial, depending on regulatory interactions, and the anticipated timing thereof, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Sangamo’s actual results may differ materially and adversely from those expressed. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the uncertain timing and unpredictable nature of clinical trials and clinical trial results, including the risks that therapeutic effects observed in preliminary clinical trial results, including data from kidney biopsies, will not be durable in patients and that final Phase 1/2 STAAR study data will not validate the potential safety and efficacy of isaralgagene civaparvovec and that the patients withdrawn from ERT will remain off ERT; reliance on results of early clinical trials, such as the Phase 1/2 STAAR study, which results are not necessarily predictive of future clinical trial results, including the results of any Phase 3 trial of isaralgagene civaparvovec; the research and development process, including the enrollment, operation and results of clinical trials and the presentation of clinical data; the effects of the COVID-19 pandemic and the impacts of the pandemic on the global business environment, healthcare systems and business and operations of Sangamo, including the initiation and operation of clinical trials; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products and product candidates; the commercialization of approved products; the potential for technological developments that obviate technologies used by Sangamo in isaralgagene civaparvovec; Sangamo’s lack of resources to fully develop, obtain regulatory approval for and commercialize its product candidates; and those risks and uncertainties described in Sangamo's filings with the