Given the size of the potential prevention of Major Adverse Cardiovascular Events (MACE) market, the CSL market cap may have even increased by more than $7 billion if the phase 3 MACE trial was successful but it doesn't mean Mesoblast's market cap will replicate this $14+ billion dollar market cap increase if an AA is considered for Rexlemestrocel-L by the FDA in even a restricted cohort (say) for ischemic diabetics ....
...... one also needs to consider the changes in the FDA AA process that were introduced in 2022 before we get over excited - a lot of hurdles / steps to consider in the AA process if the FDA insist they want them all implemented prior to granting an AA, which would also need a CHF partnership to fund everything. We know the FDA like to make Mesoblast jump through every hurdle so one could anticipate they will do the same with any CHF AA considerations
Where are we at ? Who knows but Mesoblast have indicated the proposed CHF FDA meeting that has been postponed multiple times "will" actually take place this quarter so the feedback from this meeting will determine the pathway forward and subsequently the ultimate partnership outcome for this indication...... all things considered, perhaps people should temper their expectations in relation to the potential market cap even though CSL's failure yesterday could sway the FDA thinking now - just sayin'
Reforms to accelerated approval of new medicines: long overdue
In June, 2022, the US House of Representatives passed the first legislative reforms to the accelerated approval pathway since its creation by the US Food and Drug Administration (FDA) in 1992 during the HIV/AIDS epidemic. 1
Although the FDA has increasingly used accelerated approval (with 278 accelerated approvals from 1992 to 2021), this pathway has been controversial, particularly because of these drugs' high cost, uncertain efficacy, and absence of completed confirmatory studies. For example, aducanumab received accelerated approval for Alzheimer's disease despite a negative pivotal trial and the advisory committee voting against approval. This accelerated approval resulted in a 14% initial increase in Medicare Part B insurance premiums this year. 2
In exchange for earlier market access, sponsors are expected to conduct post approval studies to confirm clinical benefit. However, most post approval studies have been delayed or not completed, and, until recently, accelerated approval was not often withdrawn when confirmatory studies failed. 3
In a recent initiative, sponsors of several cancer medicines have voluntarily withdrawn accelerated approvals with negative post approval studies. 4
Without such cooperation from the sponsor, withdrawal of accelerated approval by the FDA has been protracted and exceedingly rare—bevacizumab for metastatic breast cancer is the only non-voluntary withdrawal to date. 5
The Food and Drug Amendments of 2022 grant the FDA explicit legal authority to require initiation of confirmatory studies prior to accelerated approval, setting of enrolment targets, and biannual (twice yearly) reporting of study progress. The legislation also codifies an expedited withdrawal procedure that can be triggered if the sponsor fails to conduct the required post approval study, the confirmatory study does not confirm benefit, other evidence shows the product is not safe and effective, or the sponsor disseminates false or misleading promotional materials.
MSB Price at posting:
27.0¢ Sentiment: Buy Disclosure: Held
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