Benzinga | May 3, 2021 09:42AM EDT

Avrobio Plans Registration Trial With Kidney Biopsy Endpoint To Support Potential Full Approval Of Fabry Disease Gene Therapy

* Avrobio Inc (NASDAQ: AVRO) has provided an update on its regulatory plans for AVR-RD-01, its lentiviral gene therapy for Fabry disease, currently in FAB-GT Phase 2 trial.

* In March, the FDA granted full approval to Sanofi's SA (NASDAQ: SNY) Fabrazyme (agalsidase beta) after it received accelerated approval based on a surrogate endpoint of reduction of GL-3 inclusions in biopsied renal peritubular capillaries (PTCs).

* The conversion of Fabrazyme to full approval limits the accelerated approval pathways available for new therapies to treat Fabry disease.

* Hence, Avrobio can no longer pursue an accelerated approval pathway for AVR-RD-01 and instead plans to discuss with FDA a registration trial with a primary efficacy endpoint of clearance of GL-3/Gb3 inclusions in biopsied renal PTCs.

* Registration trial is expected to initiate in mid-2022.

* To support the use of AVR-RD-01 in a broad Fabry disease population, the company expects to include female patients, eliminate antibody status exclusions and collect additional cardiovascular and CNS data in its ongoing FAB-GT trial.

* Price Action: AVRO shares are down 5.75% at $11.01 in the premarket trading on the last check Monday.