Fabry Disease Market: New Therapies Poised to Reshape Treatment Landscape
The Fabry disease market is expected to expand with the introduction of new therapies, including gene therapies and substrate reduction therapies. Several new approvals are anticipated by 2026.
The Fabry disease market is set for significant evolution driven by a pipeline of emerging therapies. Current treatment strategies primarily involve enzyme replacement therapies (ERTs) and pharmacological chaperone therapy. Approved options such as agalsidase beta (Fabrazyme), agalsidase alfa (Replagal), and migalastat (Galafold) aim to address the enzyme deficiency and manage disease manifestations.
Several promising mid- to late-stage candidates are advancing, including substrate reduction therapies (SRTs) and gene therapies. Sanofi Genzyme's venglustat and Sangamo Therapeutics' ST-920 (a gene therapy) have shown encouraging results in clinical development. Idorsia Pharmaceuticals' lucerastat is another SRT candidate nearing potential market entry.
Gene therapies, like Sangamo Therapeutics' ST-920, are anticipated to transform Fabry disease management by offering more durable benefits. ST-920 has received PRIME eligibility from the EMA and is progressing towards a Biologics License Application submission in the U.S., with potential approval expected in the second quarter of 2026.
Lucerastat, an oral SRT, is also in late-stage development, with a U.S. approval potentially by 2030. DelveInsight projects the Fabry disease market across seven major markets was valued at approximately USD 1.7 billion in 2025 and is expected to grow substantially through 2036. This growth is attributed to the adoption of novel treatments, increased disease awareness, and advances in diagnostics.
The introduction of these next-generation therapies is expected to enhance patient outcomes and quality of life by providing more effective and convenient treatment options for Fabry disease.