Genglycos (pariglasgene brecaparvovec) for Glycogen storage disease type 1a

Approved

The FDA approved this application. Approval is the starting line, not the finish: our guide to what happens in the 90 days after a rare disease approval walks through specialty pharmacies, insurance review, and how families shorten the wait.

What is being decided

Ultragenyx has a BLA under FDA review for Genglycos (pariglasgene brecaparvovec) in Glycogen storage disease type 1a. Accelerated approval for an AAV8 gene therapy.

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Frequently asked questions

When will the FDA decide on Genglycos?

The FDA has already decided: the application was approved. See the outcome details above.

What is Genglycos being reviewed for?

Ultragenyx submitted a BLA for Genglycos in Glycogen storage disease type 1a. Accelerated approval for an AAV8 gene therapy.

What happens after the Genglycos decision?

If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.

Where this date comes from

The FDA does not publish PDUFA dates; companies disclose them. This one comes from CheckRare. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified weekly.