For what it's worth; encouraging feedback from my MD cousin who regularly serves on FDA panels
Hi everyone. This is a brief story about a conversation I had recently with my very talented cousin; he is an MD and a professor at a major US university and medical school, and is deeply involved in research. The family is very proud of him for all he has achieved and for what he does for mankind in his work. He regularly serves on FDA drug review panels. He's a very strong researcher in a university hospital. I decided to ask him what he thought of Eteplirsen's chances for approval. He didn't know about the drug or about Sarepta, so I gave him the key facts. BTW, he is not specialized in anything related to DMD so his feedback was based on his experience with the FDA in general.
His response based on the basic facts that we all know was along the following lines (paraphrasing):
"The drug will be approved. If what you tell me is true, the FDA will give the drug accelerated approval. The threshold for approval for drugs for orphan drugs is significantly lower than for other drugs. The main issue here is getting the drug to the community that very much needs it and will benefit from it."
I obviously was very pleased with this feedback, and wanted to share it with you for what it's worth.
Good luck to the SRPT longs and God bless the kids with DMD.
Thanks for sharing this Encore! The FDA is on the side of the kids and will do what's right! Heck, they're human too and all logic points to granting AA. Plus the law allows and encourages granting AA in situations like this.