UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
The FDA is standing by the decision of its reviewers to require a sham-controlled trial before it will review uniQure’s Huntington’s disease therapy AMT-130, with a senior official disputing the company’s characterization of the study’s specifics.
“We asked uniQure to take their product and randomize patients to getting the treatment the way they give the treatment, which requires a skin incision, a hole in the skull and an intracranial injection of the product,” the official told reporters on a call on Thursday, according to reporting from Fierce Biotech.
Fierce did not name the senior FDA official per the request of the Department of Health and Human Services.
The official rejected uniQure’s concerns about ethics. On an investor call on Monday, uniQure’s chief medical officer Walid Abi-Saab said that a sham-controlled study would be “difficult to justify” because it would require patients to go under anesthesia for up to 12 hours and receive a superficial [burr] hole in the skull, in their skulls.
“All of these elements represent risk for these patients,” Abi-Saab said.
The official refuted this on Thursday’s call. “We did not ask them to compare against a partial sham burr hole,” they told reporters, saying that instead, uniQure would need to “anesthetize the patient and put one to three nicks in the scalp.”
AMT-130 is a gene therapy delivered surgically directly into the brain to treat Huntington’s, a rare, fatal neurodegenerative disease. UniQure backed its application with pivotal Phase 1/2 data showing that the candidate significantly slowed disease progression by 75% compared with external controls.
Analysts at Stifel said at the time that these data “clearly exceed expectations.” However, the FDA in November 2025 told the company that it “no longer agrees” that the. Phase 1/2 study would be “adequate to provide the primary evidence in support of a BLA submission” for AMT-130. UniQure called this a “key shift” from previous guidance it had received from the agency, which it claimed had previously signed off on its program design.
The senior official on Thursday’s call rejected this characterization of events, too, insisting that the FDA had asked for internal controls since the beginning, in line with “long-standing policy for at least two decades.”
“Did the FDA ever agree to accept this distorted comparison? No,” the official said, according to Fierce. “I’ve asked our people to look for any written or verbally transcribed or minutes of a meeting that document such a promise, and there is no such promise made.”
