BioMarin Quarterly Update to the Duchenne Community

CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community.   It covers important information about next week’s FDA Advisory Committee meeting and other key updates from the company.

November 2015

BioMarin: Quarterly Update to the Duchenne Community

Welcome to the second of the BioMarin quarterly updates. As you can imagine, it has been an incredibly busy time at BioMarin. Drisapersen, our lead exon skipping compound to treat Duchenne Muscular

Dystrophy (DMD) amenable to exon 51 skipping,is currently under review by the US Food & Drug Administration (FDA) and the European Medicines Agency (EMA).

Drisapersen Regulatory Updates

We are thrilled by the progress we have made on the regulatory milestones for drisapersen in the 10 months since we completed our acquisition of Prosensa.

United States

• FDA Advisory Committee for drisapersen is scheduled for November 24, 2015 and the Prescription Drug User Fee Act (PDUFA) target date for a review decision for drisapersen is December 27, 2015
  • More information about the advisory committee meeting itself can be found here: http://www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm
  • The FDA’s purpose for an advisory committee is to obtain independent expert advice on a marketing application.
  • The FDA’s responsibility is to protect public health. Consequently, FDA is required to scrutinize all data and evidence to ensure it makes the best decision regarding every marketing application.
  • The FDA documents each individual issue and potential concern in the briefing materials ahead of the advisory committee to ensure the advisory committee addresses all of FDA’s questions and potential concerns. Therefore, it is normal and standard practice for FDA’s briefing materials to include potential concerns and questions about the data for drisapersen. This document, which is called an FDA briefing book, will be released to the public on Friday, November 20.

BioMarin is looking forward to a robust discussion and is well prepared to respond to all of FDA’s questions and potential concerns. We welcome the advisory committee process because it is essential and beneficial for the medical and patient communities. Being first,scientifically and medically, comes with a responsibility to thoroughly review the data so others can learn. We focus on proven science and robust data to demonstrate safety and efficacy and are committed to making a big difference in the lives of those living with Duchenne.

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