RARE Stock Up 13% on FDA Approval of Sanfilippo Syndrome Gene Therapy
Ultragenyx's stock rose 13% following the FDA's approval of Fayuvi, the first treatment approved for Sanfilippo syndrome Type A. The approval was supported by durable clinical data.
Why it matters — This marks a significant regulatory milestone for Ultragenyx, giving the company its first approved therapy for Sanfilippo syndrome Type A and a new commercial product in its pipeline.