In summary, based on initial conversations with FDA, AVROBIO anticipates initiating a single registration trial with a kidney biopsy endpoint in mid-2022 to support potential full approval of AVR-RD-01 as first-line therapy, subject to FDA discussion and agreement. In addition, AVROBIO will not be seeking FDA accelerated approval as this is no longer a viable option due to the recent full approval of Fabrazyme® (agalsidase beta)
Two additional patients have been dosed in the last two months in the ongoing FAB-GT Phase 2 trial; AVROBIO plans to enroll a total of up to 14 participants in the current clinical trial
To support use of AVR-RD-01 in a broad Fabry disease population, AVROBIO plans to amend the FAB-GT trial protocol to include female patients, eliminate antibody status exclusions and collect additional cardiovascular and CNS data
Avrobio is available for phone calls or videoconferencing at any time. They will continue to keep you informed of their progress and any changes.
Below is the link to the press release: https://www.businesswire.com/news/home/20210503005315/en/