Spruce Biosciences Inc. (SPRB) reported positive outcomes from two recent Type B meetings with the U.S. FDA regarding its planned biologics license application (BLA) for tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo syndrome type B.
Sanfilippo syndrome type B (MPS IIIB) is a rare, inherited pediatric neurodegenerative disorder caused by a deficiency of the NAGLU enzyme, leading to the buildup of heparan sulfate in the brain.
The company held a meeting in December 2025, which focused on clinical data and regulatory strategy. The FDA confirmed that integrated data from interventional studies and natural history datasets could potentially serve as an adequate and well-controlled study to support the agency's review of TA-ERT's effect on cerebrospinal fluid heparan sulfate non-reducing end (CSF HS-NRE).
The agency also agreed that CSF HS-NRE may serve as a reasonably likely surrogate endpoint to support an accelerated approval pathway, while providing recommendations to strengthen the surrogate evidence in the upcoming BLA.
A second meeting in January 2026 addressed chemistry, manufacturing and controls (CMC) requirements. The FDA requested one drug product process performance qualification (PPQ) batch at the time of BLA submission and data from a second PPQ batch prior to mid-cycle review. To accommodate this requirement, Spruce now anticipates submitting the BLA in the fourth quarter of 2026.
Spruce and the FDA also aligned on initiating the required confirmatory study during the BLA review period. The company emphasized that long-term clinical data show reductions in CSF HS-NRE associated with meaningful benefits across cognition, communication, and motor skill acquisition in patients with MPS IIIB, a fatal pediatric neurodegenerative disorder with no approved therapies.
Chief Executive Officer Javier Szwarcberg highlighted the constructive engagement with the agency and noted that the reauthorization of the Rare Pediatric Disease Priority Review Voucher program through 2029 restores a key incentive for developing therapies for rare pediatric conditions, potentially benefiting TA-ERT if approved.
The company had a 1-for-75 reverse stock split on July 8, 2025.
SPRB closed Tuesday's trading at $58.25, down 12.14%. In pre-market trading on Wednesday, SPRB rose to $71.00, up 21.90%.
For comments and feedback contact: editorial@rttnews.com
Business News
May 08, 2026 15:50 ET Manufacturing and services sector survey results and labor market data from main economies were the highlight on the economics news front this week. Factory orders and jobs report dominated the news flow in the U.S. Similarly, industrial production data from German garnered attention in Europe. In Asia, purchasing managers’ survey results from China and the central bank decision from Australia were in focus.