What is the holdup?

There was a key hearing with FDA Commissioner Hamburg on Thursday, March 27th, before the U.S. Appropriations Committee  – the Committee that funds the FDA.

Click here to watch the full hearing.

During the hearing, Congressman Sanford Bishop (D-GA)  asked FDA Commissioner Margaret Hamburg: “In the weeks worth of data found by researchers and scientists [studying eteplirsen], there is not an issue of efficacy, and scientists say they have never seen results like this. These kids face certain death, and the longer they wait the more muscle movement they lose…. [W]hat is the holdup? And when can I tell my constituents they are going to have Eteplirsen approval?”

Commissioner Hamburg’s response: “This is an important arena, especially in Duchenne Muscular Dystrophy, and there are advances in science if we can really develop the drug for the underlying mechanism of disease….we are taking this seriously and looking at it closely.”

Commissioner Hamburg, if the FDA was really taking this seriously, you would have granted Accelerated Approval for Eteplirsen long ago…The time is NOW.

100,000 people have signed our petition to President Obama asking that the FDA do its job and advance safe + effective treatments for Duchenne.  The FDA still has not acted.

Please thank Congressman Bishop by tweeting to him here on Twitter using his Twitter handle @SanfordBishop and/or thank him here on Facebook for standing up for Duchenne families and demanding answers from the FDA!

Final convo