Genzyme’s Cerdelga NDA receives US FDA priority review designationpharmabiz.comThe US Food and Drug Administration (FDA) has granted a six-month Priority Review designation to Genzyme’s New Drug Application (NDA) for Cerdelga (eliglustat), an investigational oral therapy for adult patients with Gaucher disease
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Genzyme Announces Positive New Data from Two Phase 3 Studies for Oral Eliglustat Tartrate for Gaucher Disease
Eliglustat tartate (Genz-112638)
- Eliglustat is a new investigational phase 3 compound from Genzyme Corporation that is being studied for type 1 Gaucher Disease.
- Eliglustat works as a substrate reduction therapy by reducing glucocerebroside. formation.
- This product is an oral agent (i.e. a pill) that is taken once or twice a day in contrast to an IV infusion for enzyme replacement therapy. Enzyme replacement therapy focuses on replenishing the enzyme that is deficient in Gaucher Disease and breaks down glucocerebroside that accumulates.
- The clinical trials for eliglustat tartate are sponsored by Genzyme Corporation.