banter and General Discussion, page-9189

That's where we never agree on MSB. Ryoncil is so close to approval for Pediatric - as it is.

Ryoncil' efficacy has been ticked - ODAC have voted - regardless of what you think, the vote is done and it is a resounding tick!

The only point of contention is the Potency Assay.
If the new potency assay data is not enough.
Then starting the adult trial, is an acceptance by the FDA of the new improved potency assay.

Maybe, they need to agree on a surrogate endpoint - more centred around confirmation of the Potency Assay, but that does not require the adult trial to be completed!


"The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval.

Drug companies are still required to conduct studies to confirm the anticipated clinical benefit. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug. If the confirmatory trial does not show that the drug provides clinical benefit, FDA has regulatory procedures in place that could lead to removing the drug from the market."