Ultragenyx Pharmaceutical Inc. received FDA accelerated approval for Genglycos, a gene therapy designed to treat glycogen storage disease type Ia [1, 3].
This approval marks a significant milestone for patients with the ultra-rare metabolic disorder, as it provides the first treatment that targets the underlying cause of the disease [1, 2].
The therapy, also known as DTX401 [1], is the fifth approved medicine for the company [1]. The FDA granted the accelerated approval on Aug. 20 [2, 4].
Ultragenyx produces the therapy at its facility in Bedford, Massachusetts [2]. The company's stock rose following the announcement of the regulatory nod [6].
According to the Boston Business Journal, "Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) won accelerated approval for Genglycos, a new gene therapy made to treat an ultra‑rare genetic metabolic disorder called glycogen storage disease type Ia" [2].
The company continues to manage its pipeline of rare disease treatments. While Genglycos moves toward commercialization, other assets remain under regulatory review [3].
“Genglycos provides the first therapy that targets the underlying cause of the ultra‑rare metabolic disorder.”
The use of accelerated approval allows the FDA to expedite the availability of drugs for serious conditions with unmet medical needs. For Ultragenyx, this represents its first successful gene therapy approval, diversifying its portfolio beyond traditional medicines and validating its manufacturing capabilities in Massachusetts.
