Sangamo Therapeutics Gains FDA Pathway to Accelerated Approval for ST-920 Gene Therapy in Fabry Disease

Sangamo Therapeutics, Inc., a genomic medicine company, announced the outcome of a recent successful interaction with the U.S. Food and Drug Administration. This interaction has provided a clear regulatory pathway to accelerated Approval for isaralgagene civaparvovec (ST-920), its wholly owned gene therapy product candidate for the treatment of Fabry disease. “Fabry disease is a debilitating condition with a significant unmet medical need,” said Sandy Macrae, CEO of Sangamo Therapeutics.

Publish Date: 22-10-2024 Source: Sangamo Therapeutics, Inc.

Fabry disease, a rare genetic metabolic storage disorder, results in the progressive failure of organs and premature death. It arises from a mutation in the galactosidase alpha gene, causing a deficiency or absence of α-galactosidase A (GALA), a lysosomal enzyme. Consequently, this deficiency impairs the breakdown of globotriaosylceramide (Gb3) and related glycosphingolipids, gradually accumulating Gb3 and glycosphingolipids within lysosomes. These accumulations are believed to have cytotoxic, proinflammatory, and profibrotic effects. Patients with late-onset Fabry disease typically manifest symptoms later in life, primarily affecting a single organ system, most commonly the heart. Nevertheless, diagnosing Fabry’s disease can be challenging due to its rarity and diverse symptoms. Over time, the disease causes progressive tissue damage, ultimately resulting in organ failure. Untreated individuals, both males and females, face a reduced life expectancy compared to the general population, with end-stage renal disease and life-threatening cardiovascular or cerebrovascular complications significantly contributing to this reduced lifespan.

Fabry disease prevalence in the USA is reported to be ~1.035 cases in every 50,000 population.

However, the current Fabry Disease treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (SIG-207, Migalastat, Lucerastat, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Fabry Disease treatment. The key companies in the advanced development stage are Sigilon Therapeutics, Amicus Therapeutics, Idorsia Pharmaceuticals Ltd., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Fabry Disease to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

About Thelansis:

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.