Debra Miller Representing Patient Advocacy Community

AdCom Sarepta Votes yes

Speech at AdCom for SRP-9001

My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.  

We recognize a central question is whether the micro-dystrophin delivered via an AAV and quantified on biopsy with study participants is reasonably likely to predict clinical benefit.   Duchenne patients with very low levels of revertant fibers have a milder disease phenotype, and therefore any increase in protein is beneficial. There is considerable peer-reviewed evidence that micro-dystrophin delivers similar long-term benefit in multiple Duchenne animal models and improved strength and timed-function tests in patients participating in these clinical trials. 

The Accelerated Approval Program allows for faster approval based on surrogate endpoints for serious conditions with an unmet medical need, like Duchenne. Clear evidence of efficacy has been seen in participating patients and we believe that the risk/benefit profile is well understood by the community. Equally as important, principal investigators in the SRP-9001 open label studies have provided testimony that the drug provides a meaningful benefit. Furthermore, the company has fully recruited its confirmatory trial, and enrolled patients are committed to completing the study. We value more data to make continued informed decisions about our children’s health. All of which supports using the Accelerated Approval pathway to get this drug to patients earlier than would normally happen. A failure to do this would bring into question the purpose of the Accelerated Approval mechanism.  

Science is progressive. We do not have the luxury of waiting for the perfect drug – it will, instead, happen through scientific evolution. One approval will lead to further improvements and better treatments in the future.  Approving SRP-9001 is a huge step forward. For the past 20 years, CureDuchenne has invested in most of the therapies that are in clinical trials today, but we have no financial interest in Sarepta. Over the past few years, CureDuchenne has identified and funded promising next-generation gene therapy technologies, including nonviral delivery and other strategies for overcoming neutralizing antibodies and barriers to redosing. SRP-9001 is the bridge that will allow for preserved muscle function and keep patients walking and living long enough to benefit from future scientific progress.  

The threat to losing function and survival is real, as the lack of dystrophin leads to muscle degeneration. Once a muscle is wasted, there is no repairing it. Any delay in approval ensures with 100% certainty, the continued progression of this devastating disease – frankly, it will be too late for many. They will have needlessly lost ambulation that will never return, and their cardiac functions – the ultimate cause of death – will deteriorate. Families facing Duchenne cannot afford any delays – their clocks are ticking. Our community has a steadfast commitment to completing the confirmatory study for the benefit of all individuals with Duchenne. Help us make this the first generation of individuals that survive Duchenne. 

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