Rare Disease Report

FDA Opens PPMD's Draft Guidance For Managing Duchenne to the Public Discussion

SEPTEMBER 04, 2014
James Radke

The Food and Drug Administration (FDA) has established a public docket to discuss issues related to developing drugs for Duchenne muscular dystrophy (DMD).

Last December 12, 2013,  during a policy meeting, the FDA agreed that Parent Project Muscular Dystrophy (PPMD) and other interested parties in the DMD community could submit for FDA consideration a proposal for a draft guidance for industry on developing drugs for DMD.

In June 2014, PPMD submitted its draft guidance to the FDA on June 25, 2014. (click here for details) and now the FDA is posting that PPMD document to seek additional guidance and public comment.

Details of the FDA notice and how to submit comments can be found here.

PPMD’s ‘Guidance for Industry Duchenne Muscular Dystrophy Developing Drugs for Treatment over the Spectrum of Disease’ can be found here.

Comments will be accepted until October 6, 2014.

In the video below, Pat Furlong, President of PPMD provides a history lesson an overview of the guidance that was being drafted at the time of this conversation (at PPMD’s Advocacy Conference earlier this year).  The final guidance can be read here.

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