Sangamo's Isaralgagene Civaparvovec Nears Accelerated Approval as Fabry Disease Market Poised to Reach $5.92 Billion by 2035
核心洞察
Sangamo Therapeutics' gene therapy ST-920 (isaralgagene civaparvovec) is advancing toward accelerated FDA approval with a BLA submission planned for the latter half of 2025, following positive Phase 1/2 STAAR trial data.
The global Fabry disease (搜索) treatment market was valued at USD 2.62 billion in 2025 and is projected to reach USD 5.92 billion by 2035, growing at a CAGR of 8.47%.
Enzyme replacement therapy remains the standard of care with 76.10% market share in 2025, while gene therapies and oral chaperone treatments are expected to reshape the treatment landscape.
Sangamo Therapeutics is on the cusp of a regulatory milestone as its investigational gene therapy isaralgagene civaparvovec (ST-920) advances toward an accelerated approval pathway with the U.S. Food and Drug Administration, a development that could fundamentally reshape the Fabry disease (搜索) treatment landscape. The company announced in March 2025 that it had reached agreement with the FDA on pursuing accelerated approval, with a Biologics License Application (BLA) submission planned for the latter half of 2025.
The momentum behind ST-920 has been building steadily. In May 2025, Sangamo reported that all patients treated in its Phase 1/2 STAAR trial (NCT04046224) had reached the FDA's required one-year milestone to proceed toward accelerated approval, with a pivotal data readout anticipated by the end of June. The FDA previously indicated that data from the ongoing STAAR study could be sufficient for accelerated approval, potentially eliminating the need for further clinical trials and expediting the therapy's market entry.
ST-920 is designed as a single intravenous infusion that aims to lower the abnormal buildup of specific fatty substances and potentially slow or stop the progression of Fabry disease (搜索). The FDA has granted the therapy Fast Track designation, Orphan Drug status, and Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring the significant unmet medical need it addresses.
A Market in Transformation
The Fabry disease (搜索) treatment market is experiencing robust growth, fueled by expanding diagnosis rates and a pipeline rich with novel therapeutic approaches. According to a report by SNS Insider, the global Fabry disease treatment market was valued at USD 2.62 billion in 2025 and is projected to reach USD 5.92 billion by 2035, growing at a compound annual growth rate (CAGR) of 8.47% during the 2026–2035 period.
DelveInsight's market analysis valued the Fabry disease (搜索) market at approximately USD 1,700 million in 2024, with the United States holding the largest share among the seven major markets (7MM) at an estimated USD 880 million. Germany led European markets at approximately USD 210 million, while Japan's market was estimated at roughly USD 150 million, representing about 9% of the overall 7MM market.
North America dominated the global market with approximately 46.21% share in 2025, according to SNS Insider, driven by high-quality healthcare infrastructure, favorable orphan drug designation policies, and strong reimbursement frameworks. The U.S. market alone was valued at USD 1.09 billion in 2025 and is projected to reach USD 2.38 billion by 2035.
Evolving Treatment Paradigm
Enzyme replacement therapy (ERT) continues to anchor the treatment landscape, commanding 76.10% of market revenue in 2025. Therapies such as Fabrazyme and Replagal, supported by over two decades of clinical evidence, remain the established standard of care. The intravenous route of administration held 66.08% of revenue in 2025, reflecting the dominance of ERT regimens administered fortnightly under clinical supervision.
However, the treatment paradigm is shifting. Chaperone therapy, led by Amicus Therapeutics' oral migalastat, is expected to register the highest CAGR during the 2026–2035 forecast period, offering eligible patients a less invasive alternative to lifelong intravenous infusions. Meanwhile, gene therapy candidates like ST-920 represent what SNS Insider describes as "revolutionary treatment that can shift the management strategy for patients suffering from Fabry disease (搜索)."
Pipeline Momentum and Regulatory Developments
The Fabry disease (搜索) pipeline has seen notable regulatory activity. In March 2026, Glafabra Therapeutics announced that the FDA granted Orphan Drug Designation to GT-GLA-S03, an investigational autologous cell therapy for classic Fabry disease designed as a potentially redosable hematopoietic stem cell–based approach to provide continuous alpha-galactosidase A (搜索) enzyme production.
In December 2024, Exegenesis Bio received FDA Orphan Drug Designation for EXG110, an innovative gene therapy targeting Fabry disease (搜索). Multiple developers continue to advance liver-directed and AAV-based gene therapy candidates, with ongoing evaluation of safety, enzyme expression, and biomarker improvements.
Key companies competing in the Fabry disease (搜索) space include Sanofi Genzyme, Amicus Therapeutics, Chiesi and Protalix Biotherapeutics, AVROBIO, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, and Takeda Pharmaceutical Company (搜索), among others.
Epidemiology and Disease Burden
Fabry disease (搜索) is a rare X-linked genetic disorder caused by mutations in the GLA gene, leading to deficient or dysfunctional alpha-galactosidase A (搜索) enzyme activity and subsequent accumulation of globotriaosylceramide (Gb3) in tissues and organs. According to NCBI data cited by SNS Insider, prevalence ranges from 1 in 40,000 to 1 in 117,000 individuals in the general population, and the condition accounts for approximately 0.5% of hypertrophic cardiomyopathy cases.
In 2024, the total number of diagnosed prevalent cases in the United States was estimated at approximately 9,200, comprising roughly 3,300 individuals with the classic phenotype and 5,860 with the late-onset phenotype. The U.S. accounted for approximately 52% of diagnosed prevalent cases across the 7MM, while EU4 and the UK combined represented about 38%, and Japan contributed roughly 10%. The disease is more prevalent in males than females in the United States.
Future Outlook
The anticipated introduction of therapies like ST-920 is expected to drive significant changes in the overall Fabry disease (搜索) market. Asia Pacific is projected to record the highest growth rate at a CAGR of nearly 9.46%, driven by rising disease awareness, improving healthcare infrastructure, and expanding rare disease screening programs in China, Japan, and India.
As Sangamo Therapeutics progresses its rolling BLA submission and the broader pipeline matures, the Fabry disease (搜索) treatment landscape appears poised for a transformation that could offer patients durable alternatives to lifelong enzyme replacement therapy while expanding the commercial opportunity for developers across global markets.