Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)
Wednesday, August 19, 2026 BiopharmaWatch Research 1 min read
Key Takeaway: Ultragenyx has received FDA approval for GENGLYCOS, marking it as the first gene therapy for Glycogen Storage Disease Type Ia (GSDIa). This approval is significant as it offers a long-awaited treatment option aimed at alleviating the care burden for affected patients. Additionally, Ultragenyx has been awarded a Priority Review Voucher with this approval.
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POSITIVE FACTORS
First-ever FDA-approved treatment for GSDIa.
Provides a new option to reduce care burden for patients.
Ultragenyx's fifth FDA approval overall.
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+94%
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Phase 2 · Hypophosphatasia
Full Press Release Details
GENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa Ultragenyx received a Priority Review Voucher upon approval Ultragenyx to host conference call on
Frequently Asked Questions
What is GENGLYCOS?
GENGLYCOS is the first FDA-approved gene therapy for Glycogen Storage Disease Type Ia.
What does the FDA approval mean for GSDIa patients?
The approval provides a new treatment option aimed at reducing the burden of care for GSDIa patients.
How many FDA approvals has Ultragenyx received?
GENGLYCOS is Ultragenyx's fifth FDA approval overall.
Did Ultragenyx receive any incentives with the approval?
Yes, Ultragenyx received a Priority Review Voucher upon approval.