Going Backwards to Go Forwards


I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and sad to see this happen.

From a business standpoint, BioMarin’s protective step makes sense. The company needs to regroup to find a better way forward. But for the Duchenne community it is a step backward.

Like you, we are concerned that the approval hurdle is too challenging, causing other companies to reconsider investing in Duchenne treatments. But we are not daunted – in our hope or our investment approach.

We appreciate the BioMarin team members who have worked tirelessly to develop this treatment, and respect their position on this action. It couldn’t have been an easy decision. BioMarin did everything possible to bring this treatment to the Duchenne community.  It’s very sad that both regulatory bodies, the FDA and EMA, did not see the benefit that so many boys have received from drisapersen.

CureDuchenne is working with BioMarin during the transition phase. Patients who are currently being treated with Kyndrisa or other BioMarin treatments on exons 44, 45 and 53 need to be in contact with their investigators at their clinical trial site about the continuation of the drug.

Today we know more about the genetics behind Duchenne than ever before. We have seen the first signs of positive treatments, and our push forward continues.

Every person with Duchenne deserves hope, a future and a cure. Thanks for standing with us and with companies like BioMarin as we join in this search for a cure.

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