Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)
The FDA granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, making it the first-ever FDA-approved treatment for Sanfilippo syndrome Type A, a fatal pediatric neurodegenerative disease. It is Ultragenyx's second gene therapy approval and sixth FDA approval overall, and the company received a Priority Review Voucher. Commercial product is expected to ship to Qualified Treatment Centers within 30-60 days, supported by the UltraCare access program, with manufacturing entirely US-based at Ultragenyx's Bedford, Massachusetts facility and Andelyn Biosciences in Columbus, Ohio.
Key figures
- Drug Name
- FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111
- Phase Of Trial
- Pivotal Transpher A trial
- Approval type
- standard full FDA approval
- Shipment window
- commercial product available to Qualified Treatment Centers within 30-60 days
- Follow up duration
- clinical data extend to up to nearly 8 years
- Patient population
- approximately 3,000 to 5,000 patients in commercially accessible geographies
- Cognitive score benefit
- 23.5 point higher Bayley-III Cognitive raw score vs natural history (p<0.0001)
- Priority review voucher
- Yes
- Gene therapy approval number
- second for Ultragenyx; sixth FDA approval overall
- Median life expectancy years
- 15
- Common adr liver enzyme increased
- 85%
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