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.
https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program@DocMcstuffins I am sure Eric Rose would be very interested to hear your explanation for the 82% reduction in deaths for the ischemic sub group in the LVAD trial in the first 12 months. To show how well this compares to normal mortality rates I have included Intermacs meta data . The accelerated approval pathway allows the FDA to decide what surrogate endpoints are validated for accelerated approval . Whilst i concede that nothing is approved until it is approved , the use of the words “pivotal trial” by Mesoblast in thier press release having just met with the FDA as part of a formal regulatory process, would suggest that the FDA have accepted the continuum of evidence from both the Dream CHF and this NIH 159 patient LVAD trial as supporting licensure for an unmet need such as end stage ischemic patients who generally involve patients less than a year to live when their ejection fraction falls below 25.
My interpretation is that Mesoblast is clearly indicating that the FDA after careful consideration have chosen to accept an AA application based , either on GI bleeding as a validated surrogate for mortality..or weaning success as a surrogate for mortality . This is hardly surprising in light of the Registry meta data for normal LVAD responses in over 65s ischemics on destination therapy…which I suggest you familiarise yourself with . These sample sizes make it clear that Rexlemestrocel is doing something very special with IL6 knockdown and strengthening cardiac muscle. How on earth do you explain this high percentage of successful weans? I take it you are also aware that Mesoblast achieved some stunning results in a previous trial with 30 LVAD patients where it was calculated there was a 93% chance of successful outcomes under a Bayesian analysis . You may well argue your usual “hypothesis generating “ script and missed primary endpoints , but Mesoblast has now achieved something pretty spectacular in five separate Heart trials..ranging from P2/3 in CHF to two LVAD trials and a Phase 1/2 in HLHS. I believe , on the basis of what is contained in the Mesoblast press release , that the LVAD indication will be the subject of an Accelerated Approval application around the start of next year resulting in a potential approval in 15 months time . An additional confirmatory trial should commence in ischemic heart failure shortly afterwards but Mesoblast should be earning substantial revenues from heart long before any confirmatory trial has concluded.
Let me be clear. I believe you are wrong to suggest that another trial will need to be done before revenues can be earned for LVAD use.
I hope that is explicit enough for you , since I believe you are lecturing everyone else without understanding that the FDA has obviously taken a great deal of time to weigh up the clinical evidence in view of the missed primary endpoints and subsequently allow an application to be made.
The next stage will be a pre BLA meeting in a month or two where outstanding points can be agreed and the market updated.
Do keep up !
https://pubmed.ncbi.nlm.nih.gov/35007505/Please do not rely on the facts or opinions expressed in the above post when making an investment decision. OP
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