UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor.
UniQure believes it’s likely the FDA will call an advisory committee meeting for its Huntington’s disease gene therapy—and the company is ready to face the independent probe.
“We welcome it,” CMO Walid Abi-Saab said during the company’s second quarter earnings call on Wednesday. UniQure also anticipates that its investigational therapy, dubbed AMT-130, could receive priority review given that the asset has been granted the FDA’s Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations.
There has been a substantial degree of “jitters” across the rare disease space, H.C. Wainwright wrote in a Thursday note, pointing to the persistent regulatory uncertainty across the sector.
Just this week, for instance, Capricor Therapeutics and Replimune faced high-stakes adcomm scrutiny for their respective assets—each of which has already faced its fair share of FDA setbacks. Capricor’s cell therapy for Duchenne muscular dystrophy cardiomyopathy deramiocel was rejected last July, while Replimune’s advanced melanoma drug RP1 has been rebuffed twice.
Deramiocel’s regulatory denial was reportedly involved a high-level disagreement at the FDA: Vinay Prasad, former director of the Center for Biologics Evaluation and Research, had unilaterally canceled an advisory committee meeting that Nicole Verdun, former director of the FDA’s Office of Therapeutic Products, had arranged for deramiocel, according to a June 2025 open letter from Integrity Advisors’ Bhavneesh Sharma. Verdun was pushed out of the agency a few weeks before Capricor received the rejection.
Capricor resubmitted its application in August 2025, after Prasad’s initial exit from the center—though he returned 10 days later. The FDA’s independent advisers this week voted in line with the original verdict: The agency’s Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3 against deramiocel’s approval, citing lack of evidence to support the effectiveness of the asset.
RP1’s rejection was similarly controversial, even prompting former FDA Commissioner Marty Makary to publicly defend the agency’s decision amid criticism. Replimune resubmitted in May, after Prasad and Makary were no longer at the agency. That gambit seems to have paid off, with the FDA’s external advisers giving RP1 a 10–3 vote of confidence on Thursday.
UniQure’s stock has been responding to these developments, showing some volatility “as investors continue to scrutinize each and every regulatory event where flexibility is part of the equation,” Stifel wrote in a note on Wednesday.
“But as we see it, the actual readthrough to QURE from CAPR or REPL is pretty limited, the firm added. Like Capricor and Replimune, uniQure has had to contend with inconsistent feedback from the FDA. Last year, the FDA said that Phase 1/2 data for the biotech’s investigational gene therapy would no longer be sufficient to support a biologics license application—despite having previously agreed to the company’s study protocol and statistical analyses.
The regulator has since walked that back and agreed that not only could three-year data from the Phase 1/2 study support an application, but also that uniQure wouldn’t necessarily need to employ a sham surgery control—a design that would involve drilling a hole into the skulls of patients who wouldn’t receive the active drug and which many have argued would be unethical. This FDA reversal similarly came after the departure of Prasad and Makary.
On uniQure’s Thursday call, CEO Matt Kapusta said the company’s recent interactions with the FDA “have been constructive and productive,” insisting that the advisory committee meetings for Replimune and Capricor do not necessarily have spillover to uniQure.
“Each program is evaluated on its own merits, on its own data, and its own patient population,” he added. “In the end, our view is that the data speaks for itself and we would very much look forward to the extent that there’s an adcomm in participating and having that discussion.”
