for Sanfilippo Syndrome Type A
Sponsor announced submission . FDA filing review (~60 days) in progress; PDUFA date estimated as acceptance + 10 months Standard Review. Card updates with confirmed dates when FDA acceptance announcement lands.
Sanfilippo syndrome type A is a rare genetic disorder where the body can't properly break down certain sugar molecules, leading to toxic buildup that damages the brain and spinal cord over time. Rebisufligene etisparvovec delivers a working copy of the faulty gene needed to process these sugars, aiming to restore the missing enzyme function. This gene therapy could potentially slow or prevent the neurological decline that robs children of speech, mobility, and cognitive abilities.
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Source: ClinicalTrials.gov