Spruce Biosciences Stock More Than Quadruples After Breakthrough Therapy Designation To Therapy For Rare And Fatal Genetic Disease

The FDA Breakthrough Therapy Designation is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions.

Spruce Biosciences, Inc. (SPRB) on Monday said that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to 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 lysosomal storage disease caused by a deficiency in the enzyme alpha-N-acetylglucosaminidase, leading to the accumulation of heparan sulfate in cells, particularly neurons. It causes progressive neurological symptoms like cognitive and developmental impairment, hyperactivity with aggressive and destructive behavior, delayed speech, hearing loss, and motor skill deficits.

Tralesinidase Alfa Enzyme Replacement Therapy is intended as an enzyme replacement therapy for the treatment of patients with MPS IIIB. The Breakthrough Therapy Designation is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions. It enables intensive FDA guidance and eligibility for rolling submission and priority review.

Shares of SPRB traded 305% higher at the time of writing. 

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