Let's hear some more from the bio industry bagholders claiming that FDA publishing CRLs is just a "nothing burger" $REPL https://t.co/QGnKP3d2Jv
$REPL my read of the RP1 CRL suggests OCE (CDER) took the lead in the review. Their objections led to the first rejection, as I previously reported, and nothing Replimune submitted for the second review was enough to support an approval. In other words, don’t blame Vinay Prasad for this one.
Replimune $REPL skin cancer drug that became FDA flashpoint is rejected again https://t.co/7aOfKy8vC7 via @Jasonmmast
$REPL FDA rejected RP1 CRL letter https://t.co/eMPqhybqHS
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
On $QURE: I wrote this in my Sept. 25 newsletter: "Bear in mind: What we have seen so far of the AMT-130 data are mentioned in a Uniqure press release. Fairly detailed, yes, but it will be important to see the data fully presented at a future medical meeting and published in a peer-reviewed journal. The validity of external control arms like the one used by Uniqure is still hotly debated, even as regulators, including the Food and Drug Administration, allow them under certain circumstances. It’s been lost to memory, but recall that the AMT-130 study started with a true, sham-surgery control arm that showed no benefit for the gene therapy. It was only after the company reconfigured the study and started using natural history comparators was a benefit observed." To be clear: I was also very positive on the data and its regulatory stance at the time. I thought this was a gene therapy that would be approved, even with Vinay in charge. That looks wrong now, unless we see another pivot a la $REPL.
FDA cancer chief Rick Pazdur blinks in his standoff against $REPL? Are there broader implications, like $SMMT or $IOBT?
Wow! $REPL - FDA accepted the resubmission of the RP1 application for advanced melanoma. PDUFA date April 10, 2026.
$REPL +25% today on the WSJ editorial bashing (again) Vinay Prasad? Super interesting. Reminder, I will be interviewing Replimune's CEO on stage at the STAT Summit Oct. 15. Joining us onstage will be $CAPR CEO. Info/tickets https://t.co/Ls3j1cYQ1p
$REPL argument to FDA not yet persuasive, apparently. As I reported earlier, Rick Pazdur's oncology team was strongly against accelerated approval, a position subsequently supported by CBER leadership, even as some reviewers pushed for approval. https://t.co/hQpakAChmZ
Quite a few (redacted) oncology CRLs released then: $REGN odronextamab x3 SC Rybrevant $JNJ $REPL $ATRA Patri-dxd $MRK $DSNKY Camrelizumab + rivoceranib x2
Hey folks - FDA has posted more Complete Response Letters, including those sent to $CAPR and $REPL. My quick read doesn't reveal any issues / deficiencies not previously disclosed. You can find the new CRLs here: https://t.co/n2VKUet580 Let me know if you see anything important that I missed.
$REPL schedules FDA meeting https://t.co/w2PwBP9JN5
The STAT Summit is Oct. 15-16 in Boston. We'd love to see you in the audience this year. To entice, we posted a preliminary agenda, with more speakers to come. https://t.co/DJjPFoIMA3 I'll be moderating a panel titled, "Biotech Faces FDA Uncertainty" featuring the CEOs of $CAPR and $REPL + one more newsworthy CEO who we hope to confirm soon. This will be a VERY topical conversation given all the FDA drama of late. My colleague @ADeAngelis_bio and I will also be onstage to chat with biotech A listers Arie Belldegrun and Noubar Afeyan about the current and future climate for biotech financing. You can register for the STAT Summit here: https://t.co/hvMIKIDdad
New from me: FDA ‘uncertainty’ forces Krystal Biotech $KRYS to shutter cancer drug trial The decision follows rejection of a similar drug developed by Replimune Group $REPL https://t.co/nY1w3BdmCd
Any decent memes out on the it's-not-Prasad-it's-Pazdur $REPL debacle?
But if I had posted the $REPL story during market hours, this person would have accused me of manipulating the stock, too. Next time, of course, I will consult with him. My bad.
FDA’s top cancer drug regulator played key role in rejection of Replimune $REPL therapy, officials say https://t.co/JYjkpdyXXu
Are we sure Vinay Prasad’s ouster is positive for biotech? Some musings in this week's newsletter. A commonly held view of his three-month tenure running the FDA’s biologics division was that he was more obstructionist and inflexible toward cell and gene therapies than one might have predicted, even accounting for his reputation as a conservative data nitpicker. The FDA rejected $REPL $RARE and $CAPR. Prasad went to war against $SRPT His departure means the FDA will revert to its more permissive, flexible ways, the thinking goes, and that’s good for developers of cell and gene therapies, good for makers of drugs for rare diseases, and a tailwind for biotech overall. I’m not so sure. You know me by now. I have a hard time fully buying into the rosy story. What if Prasad’s replacement as CBER chief is worse? By that, I mean a person who is more politically motivated and more in the MAHA, anti-pharma and anti-vaccine orbit of Health and Human Services Secretary Robert F. Kennedy Jr. Read more at link below, plus some thoughts on the road ahead for $SRPT
Replimune $REPL skin cancer therapy rejected by FDA The decision is another signal that the FDA’s top regulator of cell and gene therapies is taking a hardened stance on new drug approvals https://t.co/rBOHATLQnS
$REPL rejection follows the rejection of $CAPR and $RARE drugs, and the FDA's actions against $SRPT. CBER Director Vinay Prasad is setting a different tone than his predecessor.
From the $REPL release: The CRL indicates that the FDA is unable to approve the application in its present form. The FDA has indicated that the IGNYTE trial is not considered to be an adequate and well-controlled clinical investigation that provides substantial evidence of effectiveness. Furthermore, the FDA said the trial cannot be adequately interpreted due to the heterogeneity of the patient population. The CRL also states that there are items related to the confirmatory trial study design which need to be addressed, including contribution of components. Importantly, no safety issues were raised.
$REPL - FDA rejection https://t.co/spkkmwlEm2
$REPL halted. RP1 approval decision on deck.
#ESMOGI25 $REGN $GSK $RHHBY $COGT $REPL
