Ultragenyx Pharmaceutical Resubmits UX111 BLA for Sanfilippo Syndrome

Key Takeaways RARE resubmitted its UX111 BLA to the FDA seeking accelerated approval to treat Sanfilippo syndrome type A.Ultragenyx addressed prior CRL CMC issues, citing facility and process fixes unrelated to product quality.RARE added long-term neurologic and CSF biomarker data. FDA review could take up to six months.Ultragenyx Pharmaceutical (RARE) announced the resubmission of its biologics license application (“BLA”) to the FDA seeking accelerated approval for AAV gene therapy candidate UX111, which i ...