After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application.
The stars are aligning for previously thwarted biotechs—the latest being uniQure, which announced Wednesday that it intends to submit a biologics license application for its Huntington’s disease gene therapy in the third quarter. The move comes after the FDA agreed that three-year data from the company’s Phase 1/2 trial can support accelerated approval.
“This is obviously a ~180 from FDA,” Stifel wrote in a Wednesday morning note to investors that began with the superlative “WOW.”
Shares of the Massachusetts and Amsterdam–based biotech were up 62% at market open on Wednesday to $44 dollars following the update from its Type B meeting with the FDA. uniQure’s stock began to rise early last month on rumors of the impending departure of former FDA commissioner Marty Makary, who had been critical of the therapy.
Further, the agency will not necessarily require a sham surgery design for what uniQure in its Wednesday press release called a confirmatory study of AMT-130. The agency had previously been insisting on an additional sham surgery–controlled Phase 3 trial before it would consider AMT-130 for approval—a study design many experts and patient advocacy groups contend is unethical.
But the agency is now considering a standard-of-care control instead, according to the biotech’s announcement. Whatever details the two parties agree to, “the FDA seeks to align on the confirmatory study design prior to the BLA submission,” uniQure noted. “FDA communicated that they would work as expeditiously as possible with uniQure on this effort.”
If approved, AMT-130—which slowed disease progression by 75% after three years in the aforementioned Phase 1/2 trial—would be the first genetic treatment for Huntington’s disease.
“Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” uniQure CEO Matt Kapusta said in a prepared statement on Wednesday.
It’s been a long and bumpy regulatory road for uniQure, to say the least. The company had originally intended to seek approval of AMT-130 in the first quarter of this year, based on the same three-year data. However, during a pre-BLA meeting in November 2025, the FDA told uniQure it “no longer agrees” that data from the company’s Phase 1/2 trial are “adequate to provide the primary evidence in support of a BLA submission,” per a uniQure update at the time. This was despite the two parties having previously agreed on the protocols and statistical analyses used, according to the company.
In February, Makary appeared to malign AMT-130 in an interview with CNBC. “There was a product where the researchers drilled a burr hole, literally a hole, in people’s skulls” to directly inject a drug candidate into patients’ brains, Makary said during the interview. “At the end of the randomization period, it was found no benefit, and yet this is one of the drugs that we were pressured to approve.” While Makary did not name the product, it was widely supposed that he was referring to uniQure’s gene therapy.
UniQure is one of several companies to have previous FDA guidance reversed by the agency under the second Trump administration—a list that also includes Capricor Therapeutics and Biohaven. It is also the latest biotech to recently report alignment with the agency following Makary’s forced exit a little over a month ago.
Indeed, Stifel analysts noted that this development is similar to the alignment announced earlier this week by Lexeo Therapeutics regarding the design of a pivotal trial design for its Friedreich ataxia gene therapy. Last week, Disc Medicine announced it had reached an agreement with the FDA to resubmit for approval of its previously rejected therapy for a rare blood disease. And Moderna got positive news on Tuesday when documents released ahead of Thursday’s advisory committee meeting for the company’s mRNA-based flu shot indicated alignment with the agency.
“UniQure’s AMT-130 BLA submission highlights renewed regulatory flexibility,” William Blair wrote in a Wednesday note. “Given Vinay Prasad’s and Marty Makary’s public opinion on this filing and [complete response letter] issuance and now the positive uniQure regulatory update for AMT-130, we believe the current FDA, largely in caretaker mode, appears to be more flexible on regulatory paths for applications where concerns were previously raised.”
UniQure is also anticipating four-year data for AMT-130 in the third quarter, “which even if not the primary basis of the BLA, is likely to be important,” Stifel said in its note on Wednesday. “We’re optimistic that it should look solid.”
Correction (June 17) : The headline of this story has been updated to reflect that uniQure’s near-term submission for AMT-130 is not a “resubmission” but a first BLA. BioSpace regrets the error.
