FDA approves first-ever gene therapy for Sanfilippo syndrome — SkimNews
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- FDA approved Ultragenyx's gene therapy Fayuvi on Thursday for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as "childhood Alzheimer's."
- Fayuvi is the first drug specifically approved to treat Sanfilippo syndrome, according to the report.
- Ultragenyx, the drug's developer, did not immediately release a price for the therapy.
- Cara O'Neill, chief science officer of the Cure Sanfilippo Foundation, said the approval means families receiving a Sanfilippo diagnosis would no longer be told to "take their kids home and love them" but instead given "hope and an action plan for treatment."
- O'Neill described the disease as horrific, with affected children suffering and passing away early — the backdrop that made the approval so significant for the patient community.
Why it matters: Fayuvi's approval creates the first FDA-sanctioned treatment pathway for Sanfilippo syndrome type A, a patient community that previously had no drug options. The undisclosed price leaves affordability and insurance-coverage questions open for an ultra-rare population that has waited years for any therapeutic alternative.
Ask SkimNews
