Rare Disease Report

100,000 People Sign Duchenne Petition

MARCH 25, 2014
James Radke

Less than a month ago a group of advocates for Duchenne muscular dystrophy started a petition for the White House to encourage the  Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy,  They called themselves the Race to Yes.
In order for the petition to be officially recognized by the White House, a petition had to obtain 100,000 signatures within a specific time frame.  With 4 days to go before the May 29th deadline, the Race To Yes reached their 100,000 goal.
Rare Disease Report congratulates the Race To Yes team for a job well done. And not just for getting the petition signed but also for spreading awareness of rare diseases like Duchenne muscular dystrophy in which families often feel trapped between wanting to save their loved one from a progressive disease and being unable to get the drug quick enough because corporations are required to adhere to certain rules set by the FDA.  The Race To Yes team hopes that by allowing drugs for Duchenne to be on the Accelerated Approval Pathway, the drugs they believe are working will be approved more quickly.

What is Accelerated Approval?

To be eligible for accelerated approval, a drug must  treat a serious or life-threatening illness and provides meaningful therapeutic benefit over existing treatments.  Accelerated approval is designed to help a drug developer use a biomarker instead of a more traditional outcome measure to gain drug approval. In the case of Duchenne, they hope to use dystrophin  levels in muscle tissue as a surrogate marker for clinical efficacy instead of the more traditional 6 minute walk test.

So why won’t the FDA allow Duchenne clinical trials to use dystrophin as a surrogate marker? We are not sure but the silence from the FDA on the matter would hint that more data is needed before it can be confirmed that it is a good marker for clinical efficacy.

And while dystrophin may or may not be a good surrogate marker, one of the advantages it has over the traditional 6 minute walk test is that dystrophin levels can be measured in all Duchenne patients – not just the small percentage of boys in the patient population who are beginning to decline in their ambulation (ie, the best time to measure drug efficacy).
In a very rare disease with a limited patient population, the surrogate marker would be of great benefit. But again, only if it is an accurate assessment of clinical outcome.

The Drug Developer

One company that would greatly benefit from the Accelerated Approval would be Sarepta Therapeutics.  As we have reported in the past, Sarepta has been hoping to obtain Accelerated Approval for their drug eteplirsen. Unfortunately, that seems less likely given the recent request by the FDA to have Sarepta develop a new placebo controlled clinical trial instead of accepting the company’s current phase 2b study. Sarepta remains cautiously optimistic they can obtain Accelerated Approval for not just eteplirsen but also for the other exon skipping drugs they have in development.
But, the silence from the FDA on the Accelerated Approval option for that drug may have been one reason the Race for Yes was created. They are tired of the silence and tired of waiting.  And as a follower of the Duchenne story for several years, I know the parents of Duchenne boys are educated, organized, and tenacious.  For example – they got the White House’s attention by getting 100,000 signatures in less than a month.

So what happens next?

That remains to be seen.  Sarepta is continuing its plans to develop a placebo controlled study, the White House will look at the petition to see if it has merit, and the FDA will continue to consider Accelerated Approval if the data warrants it.  Those three things are a given.
There is also one other component that may be more interesting and more relevant to not just Duchenne but to many rare diseases.   The 100,000 people who signed the petition include many people who were not fully aware of the heartache and frustration that families dealing with progressive rare disease must endure. We at Rare Disease Report hope those new rare disease community members stay and help spread awareness. And we want to thank the Race To Yes group for introducing those people to our community.

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