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Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)
Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate
About this update from Spruce Biosciences, Inc.
[{"type":"text","content":"\nBreakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE)\n\n\nU.S. FDA Confirmed that CSF HS-NRE is a Surrogate Biomarker Reasonably Likely to Predict Clinical Benefit and Could Serve as Basis for Accelerated Approval\n\n\nBiologics License Application Submission of TA-ERT for MPS IIIB Remains on Track for the First Quarter of 2026\n\n\n SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--\nSpruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB).\n\n\n“We are pleased to receive U.S. FDA Breakthrough Therapy Designation as we prepare to submit the Biologics License Application of TA-ERT for the treatment of MPS IIIB in the first quarter of 2026. This designation highlights TA-ERT’s potentially transformative clinical impact as the first disease-modifying therapy to treat MPS IIIB in children impacted by this devastating condition,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences. “The integrated group-level clinical data demonstrates a rapid, profound, and durable effect of TA-ERT in normalizing CSF HS-NRE, the pathogenic factor leading to neurodegeneration, and stabilizing cortical grey matter volume and cognitive function in children with MPS IIIB.”\n\n\nBTD is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions where preliminary clinical evidence suggests the potential for substantial improvement over existing treatments. The designation facilitates more intensive FDA guidance, cross-disciplinary collaboration, and eligibility for rolling submission and priority review.\n\n\nAbout Sanfilippo Syndrome Type B (MPS IIIB)\n\n\nMPS IIIB is an ultra-rare, serious, and fatal genetic disease characterized by deficiency in N-Acetyl-Alpha-Glycosaminidase (NAGLU), an enzyme required for the catabolism of heparan sulfate (HS) in lysosomes. It ...