BioMarin DMD Update for Patient Groups


We wanted to share the following information from BioMarin in regards to yesterday’s news on the FDA decision on Kyndrisa (drisapersen).



Dear Patient Group Representative,

As you are likely aware, yesterday, we announced that the FDA issued a Complete Response Letter to BioMarin for our new drug application for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping. We are disappointed to let you know that the FDA has concluded that the standard of substantial evidence of effectiveness has not been met. We are reviewing the Complete Response Letter and will work with the FDA to determine the appropriate next steps regarding this application.

While we are disappointed in the FDA’s decision, we continue to believe that Kyndrisa provides a meaningful benefit to boys with Duchenne amenable to exon 51 skipping. The ongoing Kyndrisa extension studies will continue, as will the ongoing clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is exploring next steps for this application. Patients currently receiving Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on therapy.

We are incredibly grateful to the Duchenne community for its unwavering support during this process and will continue to work with you as we move forward. The patients and families that we serve are extraordinary, and we appreciate their participation in the clinical trials. The optimism, resiliency and thoughtfulness of our patients motivate us every single day.

Kind regards,

BioMarin Patient Advocacy

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