Six Emerging Therapies Poised to Transform the Fabry Disease Landscape

The current treatment paradigm, dominated by therapies like Fabryzyme and Replagal, faces ongoing challenges including infusion burdens and immunogenicity. As clinical development advances, six primary candidates are emerging to reshape patient care through 2036. Among the most anticipated is Sangamo Therapeutics’ ST-920, a liver-tropic gene therapy currently undergoing a rolling Biologics License Application filing, with US approval expected by the second quarter of 2026.

Beyond gene-based approaches, pharmaceutical companies are targeting substrate reduction to bypass the need for traditional enzyme activity. Idorsia Pharmaceuticals is advancing its oral candidate, Lucerastat, which functions independently of GLA mutation status and is slated for potential US approval by 2030. Similarly, Sanofi Genzyme is developing Venglustat, an oral inhibitor designed to cross the blood-brain barrier. Other notable entrants include UniQure’s AAV5-based AMT-191, AceLink Therapeutics’ potent AL1211, and Glafabra Therapeutics’ GT-GLA-S03, which seeks to provide a re-administrable cell therapy option. These innovations, coupled with improved diagnostic technologies, are expected to drive significant market growth by enabling earlier intervention and more durable therapeutic outcomes.