Avrobio halts rare disease program after unexpected trial data

Jan. 4, 2022

Avrobio revealed that its Fabry disease program will be deprioritized and that the company will shift focus to other clinical-stage programs in its lysosomal disorder pipeline.

According to the Cambridge, Massachusetts-based gene therapy developer, the decision to stop work on the rare inherited disorder is due to several factors; chief among them new clinical data showing variable engraftment patterns from the five most recently dosed phase 2 FAB-GT trial patients which would significantly extend the program’s development timeline. Avrobio has concluded that, due to the large degree of heterogeneity in Fabry disease, in some cases, there may be intrinsic resistance to engraftment — the process by which modified stem cells take root in the body after being reinfused —related to the unique underlying pathophysiology of untreated Fabry disease.

As such, Avrobio will stop enrollment for the FAB-GT clinical trial and continue monitoring the previously dosed patients for a total of 15 years as required by regulators. The company also noted an increasingly challenging market and regulatory environment for Fabry disease, which is likely a nod to the FDA's approval of Sanofi's Fabrazyme for the treatment of adults and kids with Fabry disease last March.

Instead, Avrobio will shift focus and funds to programs addressing Gaucher disease, Hunter syndrome and Pompe disease.