For those asking if you google "fda accelerated approval" you...

For those asking if you google "fda accelerated approval" you will find the following:

The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and that 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. These studies are known as phase 4 confirmatory trials. 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.

And from the announcement:

As there are currently no approved treatments for this life-threatening condition in children under 12, Mesoblast will urgently request a Type A
meeting with the FDA, expected within 30 days, to discuss a potential accelerated approval with a post-approval condition for an additional study.


Hopefully plain English served up on a platter answers some questions. I should add "DYOR" but perhaps 75% of you would prefer to badger people willing to do 3 minutes of reading to provide the exact information you require at any given time. I am not particularly upbeat about todays announcement but it would seem it is an unwanted bump on the road to success rather than the beginning of the end. November will be really interesting now.