Sangamo Therapeutics announced that the U.S. Food and Drug Administration has accepted its request for a rolling submission and review of the Biologics License Application (BLA) for isaralgagene civaparvovec (ST‑920), a gene therapy aimed at adults with Fabry disease. The acceptance followed a meeting with the FDA in October 2025 and confirms that the agency will evaluate the company’s proposed efficacy and safety data package under the accelerated approval pathway. The decision allows Sangamo to begin submitting the BLA later in the fourth quarter of 2025, moving the therapy closer to potential regulatory approval and market launch.
The FDA’s agreement to use the estimated glomerular filtration rate (eGFR) slope as a primary endpoint for accelerated approval was first confirmed in October 2024 and is now a cornerstone of the ST‑920 data package. Phase 1/2 STAAR study results presented in September 2025 at ICIEM2025 showed a positive mean annualized eGFR slope at 52 weeks, supporting the therapy’s clinical benefit. ST‑920 has also received Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as similar designations from European and UK regulators.
Sangamo’s financial performance in recent quarters underscores the importance of this regulatory milestone. Q3 2024 revenue rose to $49.4 million from $9.4 million in Q3 2023, driven by increased collaboration income. However, Q4 2024 revenue fell to $7.6 million, a sharp decline from $176.2 million in 2023, largely due to the termination of several collaboration agreements. The company reported a net loss of $23.4 million in Q4 2024, compared with a $60.3 million loss in Q4 2023, reflecting a modest improvement in operating efficiency but ongoing cash burn.
Chief Development Officer Nathalie Dubois‑Stringfellow emphasized that the FDA acceptance validates the company’s regulatory strategy and the robustness of the STAAR data. She noted that the positive eGFR slope results demonstrate the potential for a single‑dose therapy to provide meaningful clinical benefits beyond current enzyme replacement therapies. The company plans to initiate the rolling BLA submission later in the quarter, positioning ST‑920 as a transformative treatment option in a market projected to exceed $5 billion by 2030.
The regulatory advance is a critical step for Sangamo, but the company must still navigate significant financial headwinds. Continued collaboration income, cost control, and successful clinical development will be essential to achieve a sustainable path to commercialization. The FDA acceptance signals strong regulatory support, while the company’s financial trajectory highlights the need for additional capital and strategic partnerships to support the development and eventual launch of ST‑920.
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