$GRAL: FDA advisory committee meeting vote on GRAIL’s Galleri multi-cancer detection test, vote on benefit-risk (3 of 3 voting questions): 7-Yes 2-No 1-Abstain

Some key quotes from CDRH Director Michelle Tarver’s welcome remarks at today’s GRAIL Galleri advisory committee meeting: • “We strive to empower patients to be critical agents in their healthcare decisions. This necessitates that we equip them with the clear and accurate information they need to make informed decisions on the tools, the tests and the treatments that are being proposed to address their health conditions.” Tarver discussed CDRH’s public health mission and said supporting responsible innovation is central to that mission. She also noted the progress made in cancer screening, while pointing to remaining challenges and gaps especially for certain cancer types without screening options. She described the concept of cancer blood screening as a “genuinely promising opportunity.” • “Access to the right test at the right time can be truly transformative. However, at the same time, technologies with this kind of reach and novelty demand careful and rigorous evaluation.” • “Today we’re discussing a first of a kind medical device. We are committed to transparency in how we approach the evaluation of this device, and this advisory committee meeting is an important part of that commitment to the US public.” She highlighted two areas where the panel’s input would be especially valuable: 1) the benefit-risk assessment, and 2) if approved, how the test’s limitations and appropriate use should be communicated. She also thanked the panelists, researchers, presenters, members of the public, and public speakers sharing their perspectives, and closed by saying she looked forward to the discussion ahead.

Today’s FDA advisory committee meeting on GRAIL’s Galleri multi-cancer detection test is scheduled for 9AM-6PM ET. Live stream: youtube.com/live/83XzLN7Dbg4 Meeting materials: fda.gov/advisory-committees/… Let's hope the new showcases why these experts make the big bucks as they work through a complex set of questions.

FDA has posted briefing materials for Wednesday’s advisory committee review of GRAIL’s Galleri multi-cancer detection test.

HOW is that the FDA's walk back from commitments that were obtained from Pfizer LAST YEAR, commitments to generate evidence about the efficacy and safety of its vaccine, are not THE MAIN STORY with this year's approvals? Consider this exhibit 1 of the dysfunction of our media and political situation.

Maybe today would be a good day to ask why Pfizer’s post-COVID-19 vaccination syndrome study apparently was not initiated—and whether Pfizer and FDA still intend for it to be conducted. The study was one of four postmarketing commitments attached to FDA’s August 2025 Comirnaty approval under Marty Makary and Vinay Prasad’s new COVID-vaccine framework. It was supposed to begin by April 15, 2026. The prospective, randomized, placebo-controlled study was intended to evaluate circulating SARS-CoV-2 spike antigen and self-reported symptoms of post-COVID-19 vaccination syndrome or Long COVID at Months 1, 3, 6, and 12 in vaccine and control groups.

• First-in-class CELMoD • Accelerated approval on MRD-neg CR (new for this setting) • No public advisory committee Statute still requires either an AdComm or a written explanation in the action letter for drugs with no previously approved active ingredient. That used to be near-automatic for novel oncology mechanisms. Volume of formal AdComms has dropped steadily. The NDA was under review during a period of reduced reliance on traditional individual drug meetings, although there aresome signs of renewed AdComm activity under the new leadership. Not saying the data didn’t support approval, just that the external scrutiny step many still expect for a new class was skipped. Reasonable to ask for the stated reasons and whether the pendulum will swing back.

FDA has scheduled an Advisory Committee meeting (Sept. 23) to discuss and vote on GRAIL’s Galleri test, an NGS-based in vitro diagnostic intended to detect cancer-specific methylation patterns in cell-free DNA from a blood sample for the early detection of multiple types of cancer in adults age 50 and older

Just wanted to give a little context for this proposal. It came from watching a discussion topic at the Capricor meeting get cut short because of time. It got me thinking about all the other meetings I’ve watched where a discussion was cut short, where questions or discrepancies weren’t really resolved because the relevant analyses weren’t readily available, or where a committee member ended up trying to summarize a paper or analysis that no one else had a chance to review. There’s also the human component. By the time you get through the FDA and sponsor presentations, lunch, and the Open Public Hearing, the committee is often just beginning its deliberations. At that point, they’re already hours into the meeting, there’s naturally more time pressure, and they’re just getting to the most demanding part of the meeting. So calling it a “proposal” may have been a bit of a hook. Whether these particular ideas are the right answer or not, that’s really what I’m trying to address. I’d just like to see advisory committees have the time and information they need for full deliberation.

You gotta hand it to me, @US_FDA… this is Advisory Committee 2.0.

An FDA advisory committee today voted 9–3 No on Capricor's deramiocel for the treatment of cardiomyopathy in Duchenne muscular dystrophy (DMD). Voting question: “Does the available evidence provide substantial evidence of effectiveness of deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy?” Below is the gist of each committee member’s voting comment, based on my contemporaneous notes. Overall, the No voters cited an overall lack of adequate demonstration of efficacy. The "fragility" of the efficacy results was a common theme. Coffey (biostatistics) — No Too many questions surrounding LVEF, although some of the other outcomes may have been more favorable. Cassidy (patient representative) — Yes Based on the sponsor’s data and analyses, which she felt were not adequately discussed. He questioned the reliability of FDA’s report and whether the data had been properly evaluated, and believes FDA should exercise its regulatory flexibility. Dunn (patient representative) — Yes Not completely confident in the evidence, but patients have no alternatives and are willing to accept risk. Tifft (NIH; gene therapy and metabolic medicine) — No The indication was narrow and the data were not compelling enough. She said the decision was painful because a broader voting question incorporating upper-extremity function and outcomes raised in patient testimony might have produced a different answer, particularly with a larger study and perhaps with different endpoints. Wittes (biostatistics) — No “Fragile” seemed to be the word of the day. Differences across imputation methods are not unusual, but she would have liked to see more exploration of the assumptions concerning participants with missing data. Teerlink (cardiology) — No The sponsor’s own analysis did not meet the endpoint, with a p-value of 0.09, making the cardiomyopathy question relatively straightforward for him. He was also concerned that increasing LV volume could indicate harm. Earlier in the meeting, Capricor clarified that 0.09 is the corrected value reflected in The Lancet. As I understood it, the earlier value relied on an outdated approach under FDA guidance. Dodd (biostatistics) — No Agreed with reasons already stated. The endpoint may be prognostic, but there is no evidence that it is a validated surrogate. A p-value of 0.09, considered alongside the missing-data issues, was concerning. Fischbeck (hereditary neurological diseases and neurogenetics) — No Concerned about the reliance on post hoc analyses and did not want to make an unproven treatment available. Pavlakis (pediatric neurology) — No Agreed with the preceding comments: “The statistics didn’t work,” the findings were fragile, and the cardiac measure was not a sufficiently meaningful biomarker or clinical marker. Vidovich (interventional cardiology) — No A difficult decision given the unmet need, but the necessary evidence was not there. The treatment might prove effective in a different trial with different endpoints, and he believed the overall approach may still work. Note: Vidovich was not listed on FDA’s posted draft roster and appears to have participated as a late substitution for Wu. Stüve (neurology) — No Remained unconvinced. Snyder (neurobiology, cell biology and pediatric neurology) — Yes Said he might have abstained, but joked that someone had said only cowards abstain. His “scientist” vote was no; but his “clinician” vote is a qualified yes. He did not want to close the door on the therapy and supported continued research, including further assessment (perhaps in phase 4) and more work on mechanism of action and additional cardiac readouts. These are paraphrases intended to capture the gist of the voting explanations based on my notes, not exact quotations. For each member’s complete comments and full context, listen to the voting discussion directly. $CAPR

$CAPR deramiocel FDA AdComm vote: Does the available evidence provide substantial evidence of effectiveness of deramiocel for the treatment of cardiomyopathy in patients with DMD? 3-Yes 9-No 0-Abstain

$CAPR FDA adcomm is moving to discussion and voting. FDA noted they will not revise voting questions (typically their approach), but member discussion can include input on the question as well as on any other additions. FDA added that the vote is not all that all the Agency takes into account. Livestream: youtube.com/live/SUNn6YcBhnw…

.@statnews is also live-blogging the $CAPR deramiocel adcomm statnews.com/2026/07/29/capr…