- Spruce Biosciences received **Breakthrough Therapy Designation** from the FDA for its enzyme replacement therapy, **tralesinidase alfa (TA-ERT)**, targeting **Sanfilippo Syndrome Type B**.  **[1683553_1ca599e07e044b6ea4d2df5cbeb0cf8a]**  This designation is supported by clinical data showing **rapid and durable effects** and the FDA confirmed the biomarker could serve as a **surrogate endpoint** for potential accelerated approval.  **[1683553_1ca599e07e044b6ea4d2df5cbeb0cf8a]** 
- The company plans to submit its **Biologics License Application** for TA-ERT in **early 2026**.  **[1683553_1ca599e07e044b6ea4d2df5cbeb0cf8a]** 
- Despite facing **financial challenges**, including **negative profit margins** and **risk of bankruptcy**, Spruce Biosciences maintains **strong liquidity** with a current ratio and quick ratio both at **2.6**.  **[1683553_1ca599e07e044b6ea4d2df5cbeb0cf8a]** 
- Following the announcement, **Spruce's stock surged significantly**, reflecting investor optimism about the therapy's potential.  **[1683553_1ca599e07e044b6ea4d2df5cbeb0cf8a]**

Spruce Biosciences Gains FDA Breakthrough Therapy Designation for Sanfilippo Syndrome Drug

- **Spruce Biosciences** received U.S. FDA Breakthrough Therapy Designation (BTD) for its **tralesinidase alfa enzyme replacement therapy (TA-ERT)** for Sanfilippo Syndrome Type B (MPS IIIB).  **[1683553_c2d70aef849441dd9ece815fceeda53b_0]** 
- The BTD was supported by clinical data demonstrating **normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE)**, which the FDA confirmed is a surrogate biomarker reasonably likely to predict clinical benefit and could serve as a basis for accelerated approval.  **[1683553_c2d70aef849441dd9ece815fceeda53b_0]** 
- The company remains on track to submit the Biologics License Application (BLA) for TA-ERT for MPS IIIB in the **first quarter of 2026**.  **[1683553_c2d70aef849441dd9ece815fceeda53b_0]** 
- TA-ERT is highlighted as a potentially transformative clinical impact as the **first disease-modifying therapy** for MPS IIIB, a fatal genetic disease with no current FDA-approved treatments.  **[1683553_c2d70aef849441dd9ece815fceeda53b_0]** **[1683553_c2d70aef849441dd9ece815fceeda53b_1]**

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