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

Ultragenyx · BLA
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 the FDA decided

The FDA approved Ultragenyx's BLA for Genglycos (pariglasgene brecaparvovec) in Glycogen storage disease type 1a. Accelerated approval for an AAV8 gene therapy, for patients 8 and older, to reduce daily cornstarch intake as an add-on to nutritional management.

Frequently asked questions

Has the FDA decided on Genglycos?

The FDA has already decided. It approved the application.

Details: the outcome and what it means are at the top of this page.

Source: Ultragenyx press release

What was Genglycos approved for?

The FDA approved Ultragenyx's BLA for Genglycos in Glycogen storage disease type 1a.

What to know: Accelerated approval for an AAV8 gene therapy, for patients 8 and older, to reduce daily cornstarch intake as an add-on to nutritional management.

How much will Genglycos cost?

Trial Friend does not list a price for Genglycos. What you pay depends on your insurance and on help from the drugmaker or a charity fund.

Find help: the patient assistance finder lists programs by insurance type.

What happens after the Genglycos decision?

The FDA approved Genglycos for Glycogen storage disease type 1a on August 19, 2026.

Next steps: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.

Where this date comes from

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