Spruce Biosciences, based in South San Francisco, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its therapy, tralesinidase alfa enzyme replacement therapy (TA-ERT). This designation is significant for the treatment of Sanfilippo Syndrome Type B (MPS IIIB), a rare genetic disorder with serious implications.
Impact of FDA Breakthrough Therapy Designation
The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of promising therapies that aim to treat serious or life-threatening conditions. This designation allows Spruce Biosciences to receive enhanced guidance from the FDA, making it easier to navigate the complex regulatory landscape.
Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences, expressed enthusiasm about the designation, stating that it emphasizes the potential of TA-ERT as the first effective therapy for MPS IIIB. The company anticipates submitting its Biologics License Application (BLA) for TA-ERT in the first quarter of 2026.
Understanding MPS IIIB
MPS IIIB is characterized by a deficiency in the N-Acetyl-Alpha-Glycosaminidase (NAGLU) enzyme, which is crucial for the breakdown of heparan sulfate in lysosomes. This condition affects fewer than 1 in 200,000 individuals in the United States. However, the true prevalence is uncertain due to its absence from standard newborn screening programs.
- Signs and symptoms include:
- Progressive neurodegeneration
- Cognitive impairment
- Delayed speech and hearing loss
- Motor skill deficits
- Somatic manifestations such as coarse facial features and hepatosplenomegaly
The disease typically progresses to severe dementia and loss of motor function, often leading to a life expectancy of just 15 to 19 years. Currently, there are no FDA-approved therapies for MPS IIIB, and treatment options mainly focus on improving quality of life through palliative care.
Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT)
TA-ERT is designed to restore enzyme activity in patients lacking NAGLU. It utilizes a fusion protein approach to enhance cellular delivery. This innovative method allows the therapy to be more effectively absorbed by neural cells.
Clinical trials have shown promising results. TA-ERT has been tested in three studies involving 22 individuals with MPS IIIB. The therapy has demonstrated a favorable safety profile over five years of data.
Future Prospects
Spruce Biosciences is focused on optimizing the clinical development of TA-ERT, which aims to significantly improve outcomes for children affected by MPS IIIB. As the company prepares for the upcoming BLA submission, it remains committed to advancing treatment options for this devastating condition.
For more information on Spruce Biosciences and its innovative therapies, visit El-Balad.
