Important Update: 168 Week Data Released

Dear Friends:

This week, Sarepta Therapeutics released 168 week data from its ongoing eteplirsen study.  This new data strongly confirms the safety and efficacy of eteplirsen and once again calls into question the unreasonable delays and barriers to approval raised by the Division of Neurology Products at the FDA.

According to a company press release, boys treated with eteplirsen over more than three years showed:

♦   No adverse impact nor any reportable adverse event resulting from administration of eteplirsen after more than three years in the trial;

♦   Continued stability of respiratory muscle function as assessed by pulmonary function tests. Without eteplirsen, based on a study by the European Journal of Pediatric Neurology on the natural history of Duchenne, we would expect to see a decline in pulmonary function in boys aged 12-13 (these results should, by themselves, be sufficient grounds for FDA approval of eteplirsen); and

♦   A rate of decline in muscle function that is far slower than would be expected in boys with Duchenne. Compared to boys the same age who do not have access to treatment, the boys in the eteplirsen trial have much better muscle function:


This data confirms that the sooner boys are treated with eteplirsen, the more effective the treatment will be.  In fact the four boys who were put on placebo at the start of the study consistently lag 60 meters behind the rest of the kids because they lost so much walking ability during the six months they were on placebo. Eteplirsen has demonstrated that it dramatically slows the progression of Duchenne, preserves muscle and pulmonary function, and will prolong the lives of boys with this disease.

As parents, we continue to be impressed by the data.  We would expect, given eteplirsen’s safety and efficacy, that the FDA would be working diligently to make eteplirsen available to all who can benefit as soon as possible.  Yet the FDA and its Neurology Division have instead slowed the process by adding additional time-consuming requests for data and expanded trials before allowing Sarepta to file a New Drug Application (NDA).

Our frustration grows when we hear from prominent researchers who have examined the data, like Jerry Mendell, M.D., director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital.  “With greater than three years of eteplirsen experience, the clinical outcomes that our team has demonstrated in the testing of these boys exhibit greater stability in function than anticipated at a time when we often observe a more significant decline without treatment,” stated Dr. Mendell in response to this data.  “Based on my many years of caring for patients with this devastating disease, I view these more than 3 years of data as very encouraging.”

The Duchenne community is united in its support for eteplirsen.  We call your attention to a blog post from Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, in which she states, “The 168 week data exceeds our expectations.  Eteplirsen slows the rate of progression, delays the loss of ambulation, and preserves respiratory function.” She goes on to say, “As a community, we fully support eteplirsen as a therapy… We are and continue to be believers.”

In 2012, Congress passed and President Obama signed the FDA Safety and Innovation Act (FDASIA).  The legislation gives FDA the tools and authority necessary to rapidly review and approve therapies to treat rare diseases for which there is no approved treatment.  Duchenne is such a disease, and eteplirsen is a safe and effective treatment for Duchenne.  Yet FDA continues to ignore FDASIA and erects barriers to approval.

We are asking Congress to review FDA’s compliance with FDASIA, and before they seek to pass new laws, to hold the FDA accountable for the laws already on the books.

Delay is unacceptable.  Every day, boys with Duchenne lose muscle function and breathing becomes more difficult.  Boys will not regain this muscle and pulmonary function once it is lost.

We will not stop until the race is won for all of our boys.  Thank you for all that you are doing to help us keep running.


The Race to Yes