CDER Adds Gaucher Disease to 2017 Guidance Agenda

CDER updated its guidance agenda for 2017, adding an item on Gaucher Disease.

Gaucher Disease is a rare, inherited metabolic disorder treated with enzyme replacement therapies. It is not clear what the focus of the guidance will be.

The center’s agenda was first published in January and includes more than 100 planned items, including recommendations for securing user fee waivers, REMS document formatting and details on fines for failing to meet post-marketing requirements after accelerated approval.