Ultragenyx Gains FDA Approval for Fayuvi Therapy

The Food and Drug Administration has cleared Fayuvi as the first treatment for Sanfilippo syndrome type A, a rare genetic disorder that progressively damages the brain and nervous system. This landmark decision marks a shift from merely managing symptoms to addressing the underlying course of the debilitating inherited disease.

Today, 00:37· Money Talk· 591 reads

Ultragenyx Gains FDA Approval for Fayuvi Therapy

Following the announcement, Ultragenyx Pharmaceutical shares rose 13% to close at $14.50 on Thursday, with further gains pushing the stock to $14.78 in after-hours trading. Despite this recovery, the company’s stock remains down 37% for the year, largely due to recent setbacks involving disappointing Phase 3 results for its Angelman syndrome candidate, apazunersen.

Fayuvi, formerly known as UX111, represents the company’s second gene therapy approval and its sixth overall product clearance. Ultragenyx expects to begin shipping the treatment to qualified centers within the next 30 to 60 days. In addition to the approval, the company secured a priority review voucher, providing a strategic asset for its future development pipeline.

Ultragenyx Gains FDA Approval for Fayuvi Therapy

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