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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