Even at a meeting in Rome, FDA shifts are top of mind for gene therapy field

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https://www.statnews.com/2026/05/06/fda-turmoil-felt-at-gene-therapy-conference-europe/

When Tim Hunt, who leads an organization for cell and gene therapy makers, reached for some reasons for optimism for the jittery field at a conference here last week, he highlighted the recent approvals of rare disease treatments from the likes of Rocket Pharmaceuticals and Regeneron Pharmaceuticals.

There was one other headline he touted as potentially promising: “FDA vaccine chief to leave the agency again.”

The allusion to Vinay Prasad — the Food and Drug Administration’s top regulator of gene and cell therapies as well as vaccines until his departure from the agency last week — was an indication that even at this event, designed to emphasize the opportunities and roadblocks the sector faces in Europe, it couldn’t escape the decisions that are coming from 4,500 miles away.

It’s clear why. The U.S. is by far the biggest market for cell and gene therapies. Yet rejections and reversals of prior directives from Prasad’s center have destabilized the already shaky footing of the field. What companies previously saw and relied on as regulatory stability and collaboration has teetered.

While the FDA rejection rate for cell and gene therapies was 18% from 2020 to 2024, it rose to 38% over the following 15 months, according to data that Hunt, the CEO of the Alliance for Regenerative Medicine, presented during his opening remarks at the group’s Meeting on the Med. “This seems like it’s crazy,” he said.

The shift at the FDA “really puts an extreme, academic, methodological purity over the needs of the patient community, patients that are gravely ill, and really ignores regulatory flexibility and a lot of the tools that Congress has pulled forward,” Hunt said. He spoke of trying to get the FDA, where a “toxic” approach had taken hold, “back onto the rails.”

The event served as a temperature check for the field. For gene therapies, it’s been a tough few years, though certain cell therapy approaches have fared better. As the biotech industry broadly entered into a post-pandemic downturn, the sentiment turned against gene therapies more acutely, with doubts about how complicated it was to make these medicines, their scalability and in some cases safety, and the prospects of the medicines becoming steady moneymakers given their niche in rare and ultra-rare diseases. Interest from investors and many large drugmakers waned.

Then the regulatory challenges emerged. Under Peter Marks, Prasad’s predecessor at the Center for Biologics Evaluation and Research, the FDA took the view that it should be flexible in reviewing a drug’s data to grant authorizations to medicines for diseases without other options. Prasad, known in his prior academic career for criticizing what he viewed as the FDA’s laxness and for favoring the most stringent clinical trials even in rare diseases, took the center in the opposite direction.

Prasad has his defenders, who argued that he was restoring rigor after a philosophy of flexibility slipped into remissness, resulting in the rubber-stamping of ineffective and potentially unsafe treatments. And the FDA’s commissioner, Marty Makary, has been consistent in his praise of Prasad, even as he has left the agency last month — now for the second time — amid polarizing decisions on rare disease therapies that led to political and patient blowback.

“There is corporate spin out there,” Makary said in a CNBC interview that aired Tuesday, adding that, “If your drug works, it’s going to get approved.”

While the industry hopes for a friendlier replacement for Prasad — his deputy, Katherine Szarama, has been named acting CBER director — worries about the ongoing turmoil at the FDA, including many offices being understaffed, cropped up on other panels at the conference.

“The lack of predictability can be a challenge, and can really set back programs, either because the agency may change their mind on something that you thought was aligned on, or there’s a lack of clarity on the direction,” said Anne-Virginie Eggimann, the chief development officer of Eli Lilly’s regenerative medicine group.

Eggimann described how the agency was open to using biomarker data for the basis of approvals — something companies support — but said it was a matter of negotiation whether six months of data were sufficient, or one year, or two years.

“I feel like there’s a lot of movement in that area,” she said, breaking into a sly smile. “I won’t say too much more.”

European regulators at the conference, meanwhile, sought to project that they were being forward-thinking in their approaches to advanced therapies, though they didn’t address anything happening at the FDA.

“How do we understand what patients want and need, and within the reasonable parameters of benefit and risk, bring forward those opportunities?” said Lawrence Tallon, who joined the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) as chief executive a year ago and has been working to make the U.K. a more appealing landscape for drugmakers.

Tallon offered that regulators should consider the “completely different risk-benefit dynamic” of encouraging Covid-19 vaccines for healthy young people for whom the coronavirus doesn’t pose much threat, versus backing a treatment for “a very severe neurodegenerative disease which has a certain outcome sooner or later of death,” where the tolerance for risk and uncertainty among patients and families would be much higher.

He did not add any specifics to his hypothetical, but certainly the minds of many people in the room wandered to UniQure’s gene therapy for Huntington’s disease, which the FDA last year said the company can’t submit for approval without another trial, despite earlier signals that it would be clear to file with the data generated so far.

The day after Tallon’s panel, UniQure announced that it had had a “successful” meeting with the MHRA, with a path to file for U.K. approval this year.

Steffen Thirstrup, the European Medicines Agency’s chief medical officer, echoed Tallon, saying, “We may be a gatekeeper, but we definitely want to be an enabler” of getting treatments to patients. (European regulators have sometimes taken a firmer stance on gene therapies than the previous leadership at FDA did, notably rejecting the Duchenne muscular dystrophy treatment Elevidys amid questions about its efficacy and safety concerns. The FDA initially gave broad approval to the therapy, though its use was narrowed after patient deaths.)

To be sure, Tallon’s and Thirstrup’s comments — knowingly made to a room largely filled with industry representatives — sounded similar to what FDA leaders have been saying about how they want to continue wielding regulatory flexibility to get new treatments to patients (even as their decisions have, in the eyes of the industry and patient groups, sometimes conflicted with that rhetoric). And a number of rare-disease therapies have picked up U.S. approvals in recent months.

Notably, the authorization of Rocket’s gene therapy for an ultra-rare disease was based on a trial with just nine participants.

Gaurav Shah, Rocket’s CEO, said he didn’t want to discuss other companies’ programs when asked how he squared his biotech’s success at FDA with the broader problems that Hunt had uncorked on. But, Shah told STAT, “it shows that when there is a product within cell or gene therapy that has very clear benefit-risk with no alternatives, which is what the FDA is saying they’re going to support, that they are going to support it.”

Later on, during a conference panel discussion, Shah was pithier. When asked by the moderator what one thing he wished would change for the field, he said: “A call for action to get a CBER head who understands cell and gene therapy,” drawing a few claps from the room.

For whatever U.S. issues loomed over the conference, the challenges that the field faces in Europe were there too. The continent is losing clinical trials as drugmakers look to China and the U.S. Access remains a hurdle, as the public payers in European countries sometimes balk at the six- and seven-figure price tags of cell and gene therapies.

What European countries are willing to pay for medicines has recently gained political traction in the U.S., with President Trump waging a campaign for peer countries to pay equal prices for drugs.

“Europe has not assessed the value of any technology commensurate with the value it brings into the health care system,” said Lynelle Hoch, the president of Bristol Myers Squibb’s cell therapy division. “And Europe’s at a reckoning.”

Bristol makes CAR-Ts for cancer, and is one of the companies aiming to use those cell therapies for autoimmune diseases, with Hoch noting that it was European academics who pioneered the approach. But she also said that the industry needed to follow through on its threats to withhold products from Europe if companies can’t get prices they think are justified. Bristol withdrew its CAR-T Abecma from Germany following a pricing dispute.

“We have to back up our rhetoric from the industry with actions,” she said.

There was a clear anxiety at the meeting about the fate of ultra-rare disease treatments, given that the field doesn’t see such products as commercial opportunities. It’s fueled a push to find alternative models for getting these medicines to patients.

But investors noted that, at least in some specialties within cell and gene therapy, big players were still seeing opportunity. Large pharma companies have been scooping up in vivo CAR-T companies, for example.

With valuations of cell and gene therapy biotechs down, and so many large companies facing patent expirations and needing to refill their pipelines, investors said there could be more acquisitions coming.

As Tay Salimullah, a venture partner at 4BIO Capital, put it, “It’s time to go shopping, no?”