Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-202

Many more examples of early PDUFA dates 2019- see comments re three months early- single arm trials- astoundingly high launch prices

https://www.raps.org/News-and-Articles/News-Articles/2019/11/FDA-Approves-5-New-Costly-Drugs-Well-Ahead-of-Acti

Since 21 October, the US Food and Drug Administration (FDA) has been on a tear in approving five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were expected to be approved.

For instance, FDA signed off on Vertex Pharmaceuticals’ Trikafta (elexacaftor/ivacaftor/tezacaftor), a new treatment for those with the most common cystic fibrosis mutation, after only three months of review and well ahead of its 19 March 2020 user fee action date.

She also explained how the high number of approvals in recent years for rare diseases may be influencing this perception of a lower bar, especially as more treatments are approved on the basis of a single-arm study or with an external control group. In addition, she pointed to the “astoundingly” high launch prices for some of these rare disease treatments that may also be part of the reason for the pushback.



No reason Ryoncil not approved by Si's bedside chat end of August- so I've just reloaded- missed the 90's- lets goooooooooooooooo

The FDA changed it's mind " After additional consideration" - I'm not going to sit on the sidelines- All in

Reg