FDA approves Ultragenyx's first gene therapy

Genglycos is a one-time treatment, not a daily pill. On Aug. 19, the FDA gave it accelerated approval — Ultragenyx's first gene therapy ever cleared. It treats glycogen storage disease type Ia, a rare genetic disorder. People with it can't properly store or release sugar from their liver. Left untreated, their blood sugar can crash dangerously between meals. Patients currently manage this by eating cornstarch around the clock, day and night, to keep their blood sugar steady.

In Ultragenyx's trial, patients on the new therapy needed far less cornstarch to stay stable. For some, it was close to none at all. That's a real quality-of-life change: no more setting middle-of-the-night alarms just to eat. But the price is steep. Genglycos costs $2.7 million for a single treatment, and it's only approved for patients 8 and older. Younger children, who often need the cornstarch routine most, aren't covered yet.

Keep reading — what to watch

Will insurers actually cover a $2.7 million one-time gene therapy? That question is bigger than this one disease. Ultra-rare disease treatments increasingly carry price tags like this, and insurers are still figuring out how to pay for them. Watch whether Ultragenyx or regulators expand approval to younger children next, since they're often the ones who need it most.