CureDuchenne Urges Regulators to Approve Eteplirsen, Stresses the Importance of Multiple Drugs in the Pipeline


The Duchenne community continues its fight to get an approved treatment for Duchenne.

The FDA released Briefing Document on eteplirsen, the drug for Duchenne muscular dystrophy submitted for approval by Sarepta Therapeutics Inc.  Although the review is harsh, we urge the Advisory Committee and the FDA to listen to the patient voice when making a decision.  The Advisory Committee is meeting Friday, January 22 and Duchenne parents and patients will be there in force to share their personal experiences on being on eteplirsen.

We know boys and young men with Duchenne will die without treatments, and the FDA needs to give them options. Both Sarepta’s eteplirsen and BioMarin’s drisapersen slowed or even stopped the relentless muscle wasting in at least some of the patients who used them in clinical trials. We hope the agency will be judicious and offer our sons a chance by finding an approval pathway for both eteplirsen and drisapersen.

In 2010, CureDuchenne funded AVI BioPharma (now Sarepta Therapeutics).  This early funding allowed the company to get off clinical hold and move forward to human clinical trials and the development of eteplirsen.  We are proud to have supported Sarepta early in its development of eteplirsen and we will continue to support Sarepta through the approval process.  I look forward to speaking during the public testimony portion of the meeting and sharing our perspective of what an approved treatment would mean.

We know a single drug will not cure Duchenne muscular dystrophy, which is why CureDuchenne, through its venture philanthropy, has supported a range of research efforts. We are investing in gene therapy, anti-inflammatory and anti-fibrotic drugs, and in research that would slow Duchenne’s effect on the heart muscle.

Ultimately, it will probably take a cocktail of drugs working together to cure this disease. Exon skipping, which is what eteplirsen and drisapersen are designed to do, is a cornerstone of the cocktail approach, and admittedly will require the additional therapies to reach the best outcome for patients. But, until we get the first drugs approved, we will never be able to test them as part of a combination therapy. We know the FDA must consider the safety and efficacy of any new drug, and we support that. But we know the consequences of doing nothing.

Research take years. In the meantime, drugs like eteplirsen and drisapersen can help many of our sons. Patients, parents and treating physicians believe them to be safe and effective. And they offer hope. Let’s use every weapon we have in this fight to save lives.


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