Avrobio reprioritizes pipeline programs for AVR RD 01 in Fabry disease.

The company is deprioritizing its Fabry disease program due to several factors, including new clinical data showing variable engraftment patterns from the five most recently dosed Phase II FAB-GT patients which would significantly extend the program’s development timeline, as well as an increasingly challenging market and regulatory environment for Fabry disease.

“Previously reported data from 13 patients treated across our three clinical-stage programs have shown durable engraftment out 9 to 54 months. It is the new data from the five most recently dosed Phase II FAB-GT patients that are discordant with these other data and show variable engraftment. In addition, the last 12 months have presented multiple challenging market and regulatory dynamics for our Fabry disease program, which would now be exacerbated by a meaningfully extended development timeline,” said Geoff MacKay, president and CEO of Avrobio. “We’re fully aware of the impact this difficult decision has on the patients and families whom we have had the privilege to get to know over the years, but we believe deprioritizing and halting enrollment in our Fabry disease program is the right step forward for Avrobio and preserves our ability to continue developing therapies with the potential to address urgent unmet needs in the lysosomal disorder community.”