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.

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