Ultragenyx Pharmaceutical (RARE) said Tuesday that its UX111, ABO-102, AAV gene therapy, a treatment for patients with Sanfilippo syndrome type A, has been accepted for review by the U.S. Food and Drug Administration.
In response to the company's request for accelerated approval, the U.S. health regulator has given a decision date of Aug. 18. The agency also said it is not planning to hold an advisory committee meeting to discuss the application.
UX111 is a vivo gene therapy for Sanfilippo syndrome type A, a rare fatal lysosomal storage disease with no approved treatment that mainly impacts the brain.
Ultragenyx said that the latest studies demonstrated that treatment with UX111 resulted in a statistically significant improvement in the cognition, receptive communication, and expressive communication of treated patients compared to untreated patients.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.