We are happy to share the news that the U.S. Food and Drug Administration (FDA) have published a guidance document for industry, which details what they are looking for in the development of new drugs for Duchenne and Becker muscular dystrophy.

It is encouraging to see this level of openness in the decision making process.  Any steps that can help to speed up the process of getting potential new treatments from the labs to the patients can only be of benefit to our community.

"These guidance documents provide details on how researchers can best approach drug development for certain neurological conditions – Duchenne muscular dystrophy (DMD) and closely related conditions...These guidance documents provide our current thinking and sound regulatory and scientific advice for product developers so that safe and effective treatments can ultimately be made available to patients. These documents are each a culmination of thoughtful scientific collaboration within the agency and incorporate important input from patients, researchers and advocates. We hope that providing up-to-date, clear information about our scientific expectations, such as clinical trial design and ways to measure effectiveness, will save companies time and resources and ultimately, bring effective new medicines to patients more efficiently." FDA Commissioner Scott Gottlieb, M.D.

The key areas covered in the guidance are as follows:

1. Early Phase Clinical Development Considerations

2. Drug Development Population

3. Efficacy Considerations

4. Safety Considerations

At Action Duchenne we have always been the strongest advocates of patient organisations like ourselves, regulators, pharmaceutical companies, and of course, those living with Duchenne themsleves, working together for our common aim - to cure Duchenne.  We welcome this positive step from the FDA and look forward to seeing this guidance in practice. 

A full copy of the guidance can be found here.