My email to Federal Drug Administration

A personal perspective on the U.S. Federal Drug Administration's Advisory Committee meeting for Sarepta Therapeutics exon 51 skipping Drug Eteplirsen by Alex Johnson, mother of eight year old Jack Johnson who suffers with Duchenne Muscular Dystrophy.

As I sit here waiting for my delayed flight back to London I feel close to tears. I am completely exhausted and emotionally drained. I feel like the whole trip has been a bad dream and in a minute someone is going to wake me and tell me everything is ok and the Ad Comm just didn't happen, but it did.

Having diligently read the briefing documents I didn't go into the ad comm with high hopes of a favourable review. I expected negativity but what I didn't expect was bias. I expected a fair review — surely our community deserved that, right?

During the course of the meeting it was alluded to that the sponsor, Sarepta Therapeutics, and the FDA had previously had strained relations. I can't help but feel that the bad blood between the sponsor and the FDA shaped the discussions that took place. The one thing I am sadly and frequently learning as a member of this community is that it is always the patients that suffer and lose out.

Watching the distress of the Columbus 12 boys during the final vote are memories that will haunt and stay with me for the rest of my life.

I make no attempt to shy away from the limitations of the data set presented. It was a small trial but it is important to understand that Duchenne is a rare disease and this is a drug developed from an even smaller subset of boys within the disease. It is roughly estimated that eteplirsen could benefit 13% of the Duchenne population.

I would ask that the division of neurology is made to sit and re-watch the ad comm objectively and look at the negative bias they presented through the meeting. I would also request that this analysis for bias is evaluated by an independent expert.

Although the AdComm and the FDA's final decision has no bearing on drug approvals in Europe, it sucked all the hope I had that an exon skipping drug could ever be developed in time to benefit my son from me.

I leave feeling we are stuck in a traditional drug development model where drugs typically take 10 to 15 years to develop. Time my son and the rest of the Duchenne community do not have.