FDA approves Ultragenyx gene therapy Fayuvi for Sanfilippo syndrome type A

The FDA has approved Ultragenyx's gene therapy Fayuvi for Sanfilippo syndrome type A, an ultra-rare neurodegenerative disorder sometimes called childhood Alzheimer's. MedCity News reports that it is the first approved treatment for the underlying cause of the disease.

Fierce Pharma notes the approval is a belated win for Ultragenyx, which had previously faced a regulatory rejection and a recent late-stage setback. BioPharma Dive adds that the therapy is priced at $4 million and that this is Ultragenyx's second gene therapy approval since August.

The sources agree on the core facts of the approval. They differ mainly in emphasis: BioPharma Dive focuses on pricing and commercial momentum, while STAT and MedCity News highlight the significance of a first-in-disease therapy for a condition with few options.