Amicus' Oral Fabry Drug Priced To Compete Against Traditional ERT
FDA approved Galafold under accelerated approval, paving the way for the launch of the first oral therapy to treat a subset of patients with the rare condition Fabry disease. Amicus priced Galafold at $315,000 per year.
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Rare disease specialist Amicus Therapeutics' lead product Galafold has recently launched with early success and the company's president tells Scrip further momentum could come from better Fabry disease diagnosis.
The rare disease specialist gained 10 gene therapy programs with the acquisition of Celenex, including two in clinical-stage development for Batten disease, in exchange for $100m up front. President Bradley Campbell talked to Scrip about the process that led to the deal and next steps for development.
Amicus plans to file migalastat for Fabry disease in the US in the fourth quarter, after FDA gave the greenlight, reversing a decision last year to require an additional Phase III study.