There isn't a cat in hell's chance that $ATRA gets away with just resubmitting the Allele data.
Well worth rereading the Jan 2026 $ATRA Ebvallo CRL, which lists a host of problems with the Allele study, not just the fact it was uncontrolled: unplanned efficacy analysis, high attrition rate, selection bias, confounding treatment interventions.
So, to recap $ATRA: Jan 2026: "In the new CRL FDA stated that it no longer considers the single-arm study to be adequate." May 2026: "FDA agreed that a single-arm study could serve as adequate." Shades of $REPL?
$ATRA to be continued...? https://t.co/FMBqnZ3z2v
Remember this verbatim from $ATRA, in case the FDA meeting notes ever come to light: "FDA agreed that a single arm study using an appropriate historical control ... could serve as an adequate and well controlled study."
An important update to the $ATRA / Pierre Fabre Ebvallo story just breaking: FDA to reconsider treatment for rare cancer after its surprise rejection https://t.co/8zQSb7q5jU
This week's Biotech Scorecard newsletter: Six drugmakers, six months: Charting the FDA’s course in the post-Prasad era $REPL Pierre Fabre $ATRA $QURE $CAPR $RGNX https://t.co/sGa7G6YTDn
Pierre Fabre and $ATRA said FDA meeting to discuss the Ebvallo CRL has been scheduled. (Likely occuring within the next month.)
New reporting from me on the FDA's rejection of Ebvallo, the cell therapy from $ATRA and Pierre Fabre intended for patients w/ a rare (and rapidly fatal) blood cancer. In late 2024/early 2025, FDA CBER staff examined the Ebvallo clinical data and recommended approval, pending resolution of manufacturing issues. But in January, CBER, now under Vinay Prasad, found the exact same Ebvallo clinical data deficient, and not approvable, even as the manufacturing issues were fixed. This is the inside story of a rare disease drug that was approvable, and then wasn't. It shows how the FDA's words of support for rare disease drugs are not matched by the agency's actions. Read the story here: https://t.co/tkpJQhIRM6
This week's Biotech Scorecard newsletter: The old Vinay Prasad never left. He just changed jobs Submissions to the FDA are handled by teams of reviewers, of course. But when I look across all of these recent cases, I see a consistent throwback to the old Prasad — questioning the legitimacy of biomarkers, surrogate endpoints, and single-arm clinical trials, even in the setting of rare diseases. In some of these rare-disease drug rejections, Atara $ATRA and Regenxbio $RGNX are good examples, Prasad or the staffers he directs aren’t following the agency’s own guidance. Companies are told early that their clinical development programs meet FDA requirements, but then, almost arbitrarily, the FDA changes the rules. Uniqure $QURE and its gene therapy for Huntington’s disease have been in a holding pattern for months because guidance it received long ago from the FDA is now, under Prasad’s watch, null and void. Read more at link below...
@adamfeuerstein Bear in mind also that at the point when Makary was doing this (Sunday) he already knew $ATRA had been issued a CRL. Wild! #JPM26
I guess this adds insult to injury $ATRA https://t.co/17CTRN1i2J
Statement from Pierre Fabre re $ATRA CRL https://t.co/V9fHHACgGC
Sunday: FDA tells cell and gene therapy developers not to worry about manufacturing. We got you, it's all good. We're here to help you. Marty Makary is walking through SFO shooting a video of himself to assure that THIS IS THE NEW FDA!!!! Monday: $ATRA cell therapy rejected. "... However, in a complete reversal of position by the FDA, the CRL claims that the single arm ALLELE trial, which was previously confirmed by the FDA as adequate to support the BLA filing, is no longer considered to be adequate to provide evidence of effectiveness for accelerated approval. Furthermore, the FDA stated that the trial’s interpretability is confounded due to trial study design, conduct, and analysis. The FDA’s new position is contrary to the FDA’s prior guidance to Atara, the FDA’s alignment with Atara on the clinical trial data set, and the acceptance of the trial design as a single arm study as relevant for this patient population at BLA submission. This prior alignment had been reached by Atara and the FDA through multiple, documented meetings held over the past five plus years." #JPM26
And on it goes. Really quite incredible $ATRA #JPM26 https://t.co/Da9eRRJio7
#JPM26 off to a great start for $ATRA. Looking forward to the FDA publishing the latest CRL.
Quite a few (redacted) oncology CRLs released then: $REGN odronextamab x3 SC Rybrevant $JNJ $REPL $ATRA Patri-dxd $MRK $DSNKY Camrelizumab + rivoceranib x2
Stark lessons for cell therapy players from recent $ADAP $IOVA $ATRA disasters about the need for buy-in from a wealthy partner with commercial infrastructure.
How $ATRA turned $60m into $40m
