Ultragenyx Pharmaceutical

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FDA approves first gene therapy for Sanfilippo syndrome type A

Launching Fayuvi at $3.95 million per dose; commercial shipments to qualified treatment centers expected within 30-60 days

Children with Sanfilippo syndrome type A lose the ability to walk, talk, and eventually swallow, typically dying in their mid-teens. On September 17, the FDA approved Fayuvi, a one-time intravenous gene therapy that delivers a working copy of the SGSH gene — the first treatment that changes the disease course rather than managing symptoms.

Updated Sep 19

FDA approves first gene therapy for the cause of Glycogen Storage Disease Type Ia

New Capabilities

GENGLYCOS approved and trading under the generic name pariglasgene brecaparvovec-opnr; shares rose after the news as the company awaits a second gene therapy decision.

People with Glycogen Storage Disease Type Ia can't safely go without food. Their bodies can't release stored sugar, so many eat raw cornstarch around the clock, including overnight, to keep blood sugar from crashing. On August 19, 2026, the FDA approved GENGLYCOS, the first treatment aimed at the genetic cause of the disease rather than its symptoms.

Updated Aug 20