Clarity on Subpart H

Posted by: Christine McSherry on September 22, 2013




The Duchenne community was devastated by the news released on Friday from GSK/Prosensa. As a parent and advocate, I believe this adds additional level of urgency on to an already urgent situation. While we plan on meeting with the agency, again – very soon, we are currently planning a strategy to communicate to the FDA around accelerated approval for eteplirsen and the recent announcement for a 4th biopsy.

While considering the request for a 4th biopsy – made public by the company during a presentation last week, I wanted to clarify part of the language around accelerated approval. Please note the underlined text in the paragraph below – taken from the FDA website.

Sec. 314.510 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity.
FDA may grant marketing approval for a new drug product on the basis of adequate and well-controlled clinical trials establishing that the drug product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predict clinical benefit or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity. Approval under this section will be subject to the requirement that the applicant study the drug further, to verify and describe its clinical benefit, where there is uncertainty as to the relation of the surrogate endpoint to clinical benefit, or of the observed clinical benefit to ultimate outcome.

Based on this language, I see two consequences here – first, “reasonably likely” means the correlation of surrogate/meaningful outcome does not need to be absolutely certain – and we have talked about this with the agency…but here is where I would like some clarity, “or on the basis of an effect on a clinical endpoint” does this not mean that a surrogate OR the clinically meaningful outcome can be used for accelerated approval?

I have sent my question off to Drs. Woodcock and Temple – I am curious, could eteplirsen be granted accelerated approval from the clinically meaningful outcome – the 6MWT?  Considering the risk that parents would be taking by putting their son through another biopsy – I think we need to be sure that there is no other way to grant accelerated approval, move the drug out to market and tackle the next round of exons asap.




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