Ultragenyx Pharmaceutical (RARE) said Wednesday its UX111 gene therapy led to a statistically significant improvement in cognition, receptive communication and expressive communication among patients with Sanfilippo syndrome type A compared with natural history data in untreated patients with the rare genetic disorder that affects the brain and spinal cord.
According to new data presented at a conference in San Diego, the company saw reduced levels of heparan sulfate in the cerebrospinal fluid of all patients within the first month following treatment through the August 2024 cutoff date, reporting a median drop of 65% in heparan sulfate levels and a mean duration of 34 months.
Ultragenyx filed a biologics license application with US regulators seeking accelerated approval for UX111 in December, with the US Food and Drug Administration decision and a potential US launch expected during H2, it said.
RARE shares were over 2% higher in recent trading.
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