Sanfilippo Syndrome Type B is a rare disease that affects 1 in 200,000 people in the US, causing progressive neurological symptoms and shortening life expectancy to just 15-19 years. Spruce Biosciences (SPRB) announced that its tralesinidase alfa enzyme replacement therapy (TA-ERT) has received Breakthrough Therapy Designation from the FDA.
This designation is meant to expedite the development and review of promising therapies for serious or life-threatening conditions, allowing for more intensive guidance and priority review. CEO Javier Szwarcberg stated that the company will submit an application for approval in the first quarter of 2026.
Sanfilippo Syndrome Type B causes cognitive and developmental impairment, hearing loss, motor skill deficits, and aggressive behavior. Current treatments are non-existent, drastically reducing life expectancy. However, SPRB’s TA-ERT offers hope for these patients, with shares rising over 314% at the time of writing.
The FDA Breakthrough Therapy Designation highlights Spruce Biosciences’ promising therapeutic approach to tackle this devastating disease.
Source: https://stocktwits.com/news-articles/markets/equity/spruce-biosciences-stock-more-than-quadruples-after-breakthrough-therapy-designation-to-therapy-for-rare-fatal-genetic-disease/ch6R7WfR3gl