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Received — 18 September 2026 ⏭ FDA Press Releases RSS Feed

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FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A FDA
The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.
18 September 2026 at 02:26

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

18 September 2026 at 02:26

The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.

FDA Press Releases RSS Feed
FDA Launches Expedited IND Pilot, Begins Accepting Applications FDA
The U.S. Food and Drug Administration today announced the final design of the Expedited Investigational New Drug (IND) Pilot.
15 September 2026 at 21:53

FDA Launches Expedited IND Pilot, Begins Accepting Applications

FDA Press Releases RSS Feed

By: FDA

15 September 2026 at 21:53

The U.S. Food and Drug Administration today announced the final design of the Expedited Investigational New Drug (IND) Pilot.