Spruce Biosciences Soars 1400% on FDA Breakthrough Status
FDA grants Breakthrough Therapy Designation to the company's drug for a rare, fatal pediatric disorder, paving the way for accelerated review and potential approval.
Shares of Spruce Biosciences (SPRB) skyrocketed in an astonishing 1,434% rally on unprecedented volume after the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to its leading drug candidate, tralesinidase alfa.
The designation is for the treatment of Sanfilippo Syndrome Type B (MPS IIIB), a rare and devastating genetic disorder with no currently approved treatments. Sanfilippo Syndrome is a rapidly progressing and ultimately fatal disease that primarily affects children, causing severe neurological damage. The condition belongs to a group of metabolic disorders called mucopolysaccharidoses, where the body cannot properly break down long chains of sugar molecules.
The FDA's decision to grant the special status was based on clinical data showing that the enzyme replacement therapy, tralesinidase alfa, normalized a key biomarker in the cerebrospinal fluid. The agency believes this biomarker is , potentially allowing for an accelerated approval pathway for the much-needed treatment.
"The FDA's decision marks a significant milestone for patients and families affected by Sanfilippo Syndrome Type B," the company stated in a press release. The Breakthrough Therapy Designation is designed to expedite the development and review of drugs intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
Spruce Biosciences announced its intention to submit a Biologics License Application (BLA) for the drug in the first quarter of 2026. While the regulatory nod sparked a massive rally, the company's path forward is not without challenges. According to recent analyses, , with a reported cash position of less than $17 million as of mid-2025. This indicates a pressing need to secure additional funding to support its operations and the potential commercialization of tralesinidase alfa.
Analysts have maintained a cautious 'Hold' rating on the stock, underscoring the critical importance of the company's ability to navigate the upcoming regulatory process and secure the necessary capital. In addition to the new Breakthrough designation, the drug has also received Fast Track, Orphan Drug, and Rare Pediatric Disease designations in the United States.