No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy
Noises From US FDA Positive Ahead Of PDUFA
The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.
You may also be interested in...
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.
Obstacle Thrown In Sarepta’s Path To Market As Gene Therapy Will Face AdComm After All
The route to market for Sarepta’s gene therapy candidate SRP-9001 for Duchenne muscular dystrophy has just got more complex after a U-turn by the US FDA. Its accelerated approval will now be scrutinized by outside experts – but the company remains confident in its data.
Califf: Decades Of Follow-Up Necessary For Gene Therapy
FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.