The Jett Foundation Fighting Duchenne Muscular Dystrophy

The Duchenne Blizzard Continues

February 8, 2016

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Sitting just outside of Boston Massachusetts this morning waiting for Jett to wake up, I received notification that the second blizzard of 2016 has hit.

Pun intended, south of Boston is under a blizzard warning – as is our Duchenne community.

The record breaking Washington D.C. blizzard of January 22, 2016 robbed the Duchenne community of the opportunity to have eteplirsen publicly reviewed in a transparent manner, and to testify in front of the Food and Drug Administration (FDA) and the Peripheral and Central Nervous System (PCNS) Advisory Committee (ADCOM) members in support of eteplirsen. This morning, Sarepta announced that it has been notified of a new PDUFA date – May 26, 2016, three months later than the original date of February 26, 2016. This is another damaging blow, robbing our children of more time they don’t have to give – and we cannot blame mother nature for this one.

Living in New England, I know too well that snow shovels are at times the only way to dig out of a storm. For the Duchenne community, I hope that a shovel- a tool to dig the Duchenne community out- comes in the form of an amendment to the New Drug Application (NDA), that possibly alters some of the content in the FDA briefing documents released on January 15th.

 “The FDA notified Sarepta that its January 8, 2016 submission of 4-year clinical effectiveness data, which included additional six-minute walk test (6MWT) and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. The FDA stated that the PDUFA goal date has been extended by three months to allow for a full review of the submission. As described in the Sarepta Advisory Committee Briefing Document Addendum, the principal basis for establishing the effectiveness of eteplirsen is a comparison of patients in Study 201/202 to a historical control group.”

Sarepta Therapeutics February 8, 2016 Statement on the delay of eteplirsen’s PDUFA date

Meaning, FDA is taking additional data on the 4-year progression of eteplirsen’s matched historical control under their review of eteplirsen. We know that 10 boys out of the 12 from Study 201/202 are still walking (83%), unprecedented for kids that were on a decline. We understand from the natural history data provided in the NDA that all but one of those 11 closely matched boys (9%), did exactly as expected in their progression, they stopped walking.

However, this extraordinary measure of proof of eteplirsen’s efficacy was dismissed in FDA’s briefing documents as, “The clinical course of the 12 patients participating in study 201/202 appears to be within the expected natural history of DMD.” I hope that this additional data provided to the agency could change FDA’s perspective on the historical control and its comparison to eteplirsen’s Study 201/202.

That being said, three additional months is much too long for our community to wait for the review of a safe and seemingly efficacious drug.  The effects of this delay will be measurable. There will be kids who will stop walking.  Others will lose the ability to use their arms and feed themselves. Some may die.

I only have one thing to say to FDA, an 11-month review process for a drug that treats a progressive, fatal, pediatric disease with an unmet medical need is unacceptable. There is nothing “priority” about this review. Our children deserve better then lengthy delays and erroneous briefing documents.

While it seems that FDA may finally be noticing the errors and falsehoods in their briefing documents, noticing what the Duchenne community has been telling them for nearly a month, children with Duchenne should not fall victim to this type of delay, waiting for adults to finally see the right shovels, the right tools, the right information and data, to dig them out.


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