What are chances of getting approvals of fabry gene therapy candidate of Sangamo therapeutics

The chances of getting FDA approval for Sangamo Therapeutics' Fabry gene therapy candidate, isaralgagene civaparvovec, appear high based on the following points: 1. **FDA Alignment**: The U.S. Food and Drug Administration (FDA) has provided a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, which is a positive indicator for the potential approval. 2. **Data Support**: The FDA has agreed that data from the ongoing Phase I/II STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. This means that the complete dataset to support an Accelerated Approval pathway will be available in the first half of 2025. 3. **Clinical Efficacy**: The Phase I/II STAAR study has shown encouraging safety and efficacy data, including statistically significant improvements in both mean and median eGFR levels, resulting in a positive annualized eGFR slope. These data support the potential clinical benefit of the therapy. 4. **Regulatory Flexibility**: The FDA's regulatory flexibility, as demonstrated by the agency's agreement to consider eGFR slope as a surrogate marker for kidney health, indicates a favorable review process. 5. **Industry Alignment**: The FDA's guidance aligns with the European Medicines Agency's (EMA) Priority Medicines (PRIME) eligibility, which includes enhanced regulatory support and scientific guidance. This dual alignment suggests a robust regulatory framework for gene therapies. 6. **Strategic Prioritization**: Sangamo Therapeutics has recently initiated a strategic reprioritization initiative to focus on its gene therapy programs, which includes the Fabry disease candidate. This focus could potentially lead to faster development and FDA approval compared to a more diversified pipeline. Considering these factors, the chances of getting FDA approval for Sangamo Therapeutics' Fabry gene therapy candidate, isaralgagene civaparvovec, appear strong. The company's alignment with the FDA on an accelerated approval pathway, along with positive clinical trial results, position Sangamo Therapeutics well for potential FDA approval in the second half of 2025.