The FDA will hold advisory committee meetings this week for previously rejected investigational therapies from Replimune and Capricor Therapeutics. Some industry leaders were surprised the meetings were called at all, while many have questioned the rationale behind them and other adcomms on deck.
The FDA plans to host several advisory committee meetings this summer, including for investigational therapies from Replimune and Capricor Therapeutics. After a nine-month adcomm drought, the newly announced schedule reflects an evolving agency position that is quite different from a year ago.
Still, industry and regulatory leaders wonder whether the right experts are in place to foster discussions of the highest scientific standards.
“There’s a difference between having every seat on an advisory committee filled vs. having the right people in those seats,” Peter Pitts, a former associate commissioner and senior official in charge of advisory committees at the FDA, told BioSpace.
On July 29, the FDA’s Cellular, Tissue, and Gene Therapies (CTGTAC) advisory committee will meet to discuss Capricor’s once-rejected Duchenne muscular dystrophy cardiomyopathy cell therapy deramiocel. The following day, the CTGTAC will again convene, this time to weigh the approvability of Replimune’s twice rebuffed advanced melanoma drug RP1. The agency has until two days prior to the meetings to name the adcomm members.
“It will be really important to understand who the subject matter experts that they have invited to participate are,” Nicholas Richardson, vice president of clinical development at global CRO Precision for Medicine and former FDA deputy director, told BioSpace. “It’s hard to gauge in the absence of that, because right now on the [CTGTAC] there are only four standing members.”
Moving forward, Richardson would like to see continuity of the adcomm committees’ membership, which he said will be important to maintain for the sake of scientific rigor and lessons learned over time. The chair selected for the meetings will also be important, he said.
In addition to Replimune and Capricor, Sydnexis now has a date with an FDA adcomm, for its pediatric progressive myopia candidate. Richardson said the FDA seems to be shifting course again as new shoes fill the top roles at FDA.
Former FDA commissioner Marty Makary and former Center for Biologics Evaluation and Research (CBER) director Vinay Prasad had greatly curtailed advisory committees shortly after they joined the agency in spring 2025.
“Commissioner Makary and Secretary Kennedy disrespected not just the process of advisory committees, calling the experts who serve on them shills and tools of the industry,” Pitts said. “Shame on the administration for allowing that to happen.”
But things started to shift earlier this year. AstraZeneca broke the long dry spell on May 1 with an adcomm for two cancer assets, just as Prasad exited the agency. Then Makary followed Prasad out the door, and Moderna got a meeting for its mRNA flu vaccine, which Prasad in February controversially refused to review. Now under the new temporary commissioner and center directors, there are at least three adcomms on the docket.
“I think these adcomms that are coming up within the cell and gene therapy space are a direct result of changes in leadership,” Richardson said. “[Interim Commissioner] Kyle Diamantas and [acting CBER head] Karim Mikhail have been public about their desire for more advisory committees, and these two on July 29th and July 30th are a result of that leadership.”
Pitts agreed. The agency is “regaining its balance,” he said, and recognizing that adcomms are “valuable regulatory scientific resources that help to advance public health, rather than being viewed as something to poke fun at and do away with.”
Adcomms not ‘a box to check’
The adcomms scheduled for Capricor and Replimune will revisit drugs that were previously rejected by the agency. Capricor was hit with a complete response letter (CRL) for deramiocel in July. Five months later, the cell therapy aced its pivotal trial and the FDA accepted a resubmission in March. Replimune, meanwhile, has suffered two rejections for RP1—one in July 2025, the other in April. The FDA accepted Replimune’s resubmission for the therapy in June, setting an Aug. 2 PDUFA date.
Capricor CEO Linda Marbán told BioSpace last month that she was “surprised” by the meeting’s scheduling. “We did hear that [acting FDA Commissioner] Kyle Diamantas . . . said he wanted more adcomms, and so we’re kind of wondering if this is sort of in response to that,” she said.
Marbán additionally suggested that the FDA might be more careful around DMD therapies following last year’s controversial regulatory decisions around Sarepta Therapeutics’ gene therapy Elevidys that followed the deaths of two teenage patients.
If adcomms are being scheduled for perfunctory reasons, this is something Pitts vehemently disagrees with.
“Treating advisory committees as a box to check is a huge mistake,” he said. “The advisory committees take place because there are significant issues that need discussion, and you want standing committees so they have some type of coherent awareness of the case.”
Another company to receive a surprise CRL—and adcomm—is Sydnexis. For CEO Perry Sternberg, a series of unusual circumstances surrounded the October 2025 rejection of SYD-101, the company’s formulation of atropine to treat pediatric myopia, which can cause blindness in young children.
“If you’re going to get a CRL, you kind of already know it’s coming because you’re already having questions go back and forth with the agency on topics you can agree on right then,” Sternberg said. This time, however, it was “nothing like that.”
Sydnexis filed an appeal with the FDA’s Office of Specialty Medicines, then on May 14, Sternberg and his colleagues met with then-CDER Director Tracy Beth Høeg. But they left unsatisfied, he told BioSpace. “[We] couldn’t really get an answer from the division for what their concern was.” It turned out that meeting took place on Høeg’s last day at the agency.
“She was gone,” Sternberg said. “And the next thing we know is that it went back down to the Office of Specialty Medicines and we’re going to have an advisory committee meeting.”
David Hunter, the ophthalmologist-in-chief at Boston Children’s Hospital and an advisor to Sydnexis, has been following SYD-101’s journey. He told BioSpace his first reaction—and that of the pediatric ophthalmology community—to the CRL was, “How could that be?”
The data Hunter had seen from the company’s Phase 3 trial supported approval, he said, suggesting that the rejection had perhaps come as a result of a lack of subject matter experts on the review committee.
Sternberg is looking forward to the yet-to-be scheduled adcomm meeting—as long as those experts are in the room.
“We welcome a discussion, but we need to know who’s going to be there having that rigorous scientific discussion,” Sternberg said. He has one major request for the agency: “Please get people that understand myopia around the table.”
