UniQure said on July 31 that it expects the Food and Drug Administration may call an advisory committee meeting to review its investigational gene therapy for Huntington’s disease, AMT-130, and stated it is prepared to participate in such a process.
“We welcome it,” Chief Medical Officer Walid Abi-Saab said during the company’s second quarter earnings call. UniQure also anticipates that AMT-130 could receive priority review, as the asset has been granted Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations by the FDA.
Recent regulatory uncertainty has led to volatility in the rare disease sector. H.C. Wainwright wrote in a note that there are ongoing “jitters” due to persistent regulatory uncertainty across biopharma. This week, Capricor Therapeutics and Replimune both faced scrutiny from independent FDA advisers regarding their respective therapies—Capricor’s deramiocel for Duchenne muscular dystrophy cardiomyopathy and Replimune’s RP1 for advanced melanoma—after previous setbacks with the agency.
The denial of Capricor's application last July reportedly involved internal disagreement at the FDA. According to a June 2025 open letter from Integrity Advisors’ Bhavneesh Sharma, former Center for Biologics Evaluation and Research director Vinay Prasad canceled an advisory committee meeting arranged by Nicole Verdun, then director of the Office of Therapeutic Products; Verdun left her position shortly before Capricor received its rejection. Capricor resubmitted its application in August 2025 after Prasad initially exited his role but returned ten days later. The Cellular, Tissue and Gene Therapies Advisory Committee subsequently voted 9-3 against approval due to insufficient evidence of effectiveness.
Replimune's RP1 also faced controversy surrounding its rejection but received a favorable 10–3 vote from external advisers after being resubmitted following leadership changes at the FDA. These developments have caused some volatility in uniQure's stock price as investors monitor regulatory events closely.
Stifel wrote that readthrough from these cases to uniQure is limited: “But as we see it, the actual readthrough to QURE from CAPR or REPL is pretty limited.” Like other companies, uniQure has experienced inconsistent feedback from regulators; last year, Phase 1/2 data was deemed insufficient by the FDA despite prior agreement on study protocols—a decision later reversed after leadership changes at the agency. The regulator now agrees three-year data can support an application without requiring sham surgery controls.
During Thursday's call, CEO Matt Kapusta said recent interactions with regulators have been positive: “Each program is evaluated on its own merits, on its own data, and its own patient population,” he said. “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.”
