Shares of Spruce Biosciences (NASDAQ:SPRB) jumped 60% on Monday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its investigational enzyme replacement therapy, tralesinidase alfa (TA-ERT), developed for the treatment of Sanfilippo Syndrome Type B.
The FDA designation — which accelerates the development and review process for therapies that may offer substantial improvement over existing treatments for serious diseases — was backed by long-term clinical data demonstrating normalization of cerebral spinal fluid heparan sulfate non-reducing end (CSF HS-NRE). The agency also confirmed that CSF HS-NRE may serve as a surrogate biomarker reasonably likely to predict clinical benefit, potentially paving the way for accelerated approval.
Spruce Biosciences, a late-stage biopharmaceutical company focused on developing therapies for rare neurological conditions, said it remains on schedule to submit a Biologics License Application (BLA) for TA-ERT 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, Chief Executive Officer of Spruce Biosciences.
Sanfilippo Syndrome Type B, also known as MPS IIIB, is an ultra-rare and fatal genetic disorder that affects fewer than one in 200,000 individuals in the United States. The condition results from a deficiency in the enzyme N-Acetyl-Alpha-Glycosaminidase, which is essential for breaking down heparan sulfate in lysosomes. There are currently no FDA-approved treatments for the disease, and care remains palliative.
The Breakthrough Therapy Designation provides Spruce with enhanced interaction and guidance from the FDA, cross-disciplinary collaboration, and eligibility for rolling submission and priority review, potentially speeding up the regulatory path to market for the therapy.
Spruce Biosciences stock price
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