UniQure to seek AMT-130 approval as FDA agrees on path for drug

UniQure plans to ask the U.S Food and Drug Administration (FDA) for accelerated approval of AMT-130 to treat Huntington’s disease in the next few months, after the agency dropped its request for a new clinical trial.

The agency agreed in a recent meeting that the three-year analysis from two ongoing Phase 1/2 trials — one in the U.S. (NCT04120493) and one in Europe (NCT05243017) — could support a biologics license application (BLA) seeking approval. The FDA previously said a new clinical trial should be conducted before the company could file the application.

Plans for a new clinical trial are not entirely scrapped. Accelerated approval would allow AMT-130 to be marketed based on preliminary evidence that it will benefit patients, but conversion to full approval requires additional trial data to establish that benefit.

“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,” Matt Kapusta, CEO of uniQure, said in a company press release. “We remain focused on bringing AMT-130 to patients and families as quickly and responsibly as possible in the US and globally.”

In Huntington’s, mutations in the HTT gene cause the production of an abnormal huntingtin protein that clumps and damages nerve cells, leading to movement problems and a range of other Huntington’s symptoms.

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Gene therapy aims to slow disease progression

AMT-130 is a one-time gene therapy designed to bind to an intermediated molecule derived from the HTT gene and prevent production of all forms of huntingtin, including mutant huntingtin. In doing so, it is expected to slow Huntington’s progression and ease symptoms. The therapy is delivered directly into the brain via a surgical procedure done under anesthesia.

The FDA has granted AMT-130 regenerative medicine advanced therapy, fast track, and breakthrough therapy designations, which offer various incentives to speed the treatment’s development, including increased interactions with the agency and eligibility for accelerated approval.

The ongoing Phase 1/2 trials are testing two doses of AMT-130 in people with early Huntington’s, ages 25 to 65. Their most recent findings, based on three-year data from 24 treated participants, showed that the higher AMT-130 dose slowed disease progression by 75% compared with an external group of untreated patients in natural history studies.

UniQure had planned to use that data to support an application for accelerated approval of AMT-130 based on previous interactions with the FDA. However, in a meeting last year, the agency indicated that it no longer considered the available data sufficient to support such an application.

The regulators cited concerns about using an external natural history control group to determine the therapy’s efficacy and recommended a new trial in which participants would receive AMT-130 or a sham surgery, in which participants would receive the same surgical procedure but without any active medication.

The FDA now says the three-year Phase 1/2 data can serve as the basis of an application for accelerated approval. Meanwhile, uniQure will continue working with the regulators on the design of the confirmatory study, including whether a standard-of-care control group can be used instead of a sham surgery group.

“The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study,” Kapusta said. “The consistency and strength of the clinical data generated to date give us great confidence in the product’s potential to make a meaningful difference for patients.”