SMA Foundation comments to FDA on the importance of combination therapy for rare diseases like SMA

Last month, the FDA established a public docket for comments on FDA activities performed under the Food and Drug Administration Safety and Innovation Act (FDASIA), Patient Participation in Medical Product Discussions. The SMA Foundation took this opportunity to encourage the FDA to convene a stakeholder meeting on combination therapies, with the goal of producing a strategic plan or guidance for industry on how to pursue development of combination therapies. The Foundation believes that combinations could be highly beneficial to SMA patients and to the wider rare disease community.  We hope to see the FDA take steps under FDASIA to encourage development of combination therapies.

For the Foundation’s full comment, please see the following link:!documentDetail;D=FDA-2014-N-1698-0021