Duchenne Group's Presentation Is Milestone For Patient Involvement
Advocacy group gets portion of Sarepta's formal presentation period to present eteplirsen patient experience data, believed to be a first for an advisory committee meeting. Will it become commonplace?
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Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
Sarepta Tells Patients To Stand Down, Golodirsen Future 'Our Responsibility'
Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.
US FDA Mulling Core Criteria For Patient-Reported Outcomes
Agency wants public comment on creating clinical outcome assessments that could be adaptable to multiple trials.