Ultragenyx Wins FDA Approval for GENGLYCOS, Its First Gene Therapy

FDA approved Ultragenyx’s GENGLYCOS (DTX401) for glycogen storage disease type 1A, marking the company’s first gene therapy approval. The approval is also its fifth approved medicine, a major positive milestone that should strengthen product momentum and commercial outlook.

Analysis

FDA approved Ultragenyx’s GENGLYCOS (DTX401) for glycogen storage disease type 1A, marking the company’s first gene therapy approval. The approval is also its fifth approved medicine, a major positive milestone that should strengthen product momentum and commercial outlook.