VTX-801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease
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Thursday, August 12, 2021
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Pfizer is collaborating with Vivet on the clinical supply of VTX-801 for the Phase 1/2 clinical trial.
Key Points:
- Pfizer is collaborating with Vivet on the clinical supply of VTX-801 for the Phase 1/2 clinical trial.
- The FDAs decision to grant VTX-801 Fast Track designation underscores the urgent need for new therapeutic options to address this devastating disease, which, if left untreated, can be fatal, said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizers Rare Disease Research Unit.
- VTX-801 is a novel investigational gene therapy for Wilson Disease, which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC) and Fast Track designation by the FDA.
- The trial is expected to enroll up to sixteen adult patients with Wilson Disease and will evaluate up to three doses of VTX-801.