FDA approves Ultragenyx gene therapy Fayuvi for Sanfilippo syndrome type A
The first treatment for the underlying cause of Sanfilippo syndrome type A wins approval, marking Ultragenyx's second gene therapy.
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.
Sources · 6
- FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder
- Gene Therapy for Sanfilippo Syndrome Gets FDA Nod
- Ultragenyx Gene Therapy Is First Approved Treatment for Rare Neuro Disease Sanfilippo
- Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug
- STAT+: Roche expands its Boston footprint with new research center
- STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
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