uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease

uniQure shared some exciting progress in developing a treatment for Huntington’s disease (HD). The U.S. Food and Drug Administration (FDA) has agreed on a plan to speed up the approval process for uniQure’s AMT-130 treatment.

Here’s what it means:

• Using Current Study Data: The FDA has said that data from ongoing studies (Phase I/II trials) can be compared to information about how Huntington's typically progresses in people not receiving the treatment. This data will be the main evidence for requesting an accelerated approval of the treatment.

• Measuring Success: The FDA has agreed to use a specific tool, the composite Unified Huntington’s Disease Rating Scale (cUHDRS), as a way to measure the treatment’s effects. This tool focuses on important aspects of Huntington's, like movement, thinking abilities, and daily activities.

This approval pathway could help bring AMT-130 to patients faster if the data shows it works and is safe.

Matt Kapusta, the CEO of uniQure, said that they are "going to advance AMT-130 as aggressively as possible."

Professor Ed Wild said,

For the first time, the FDA has given a clear and achievable path to accelerated approval for a Huntington's treatment. Existing trial data from AMT-130 will continue to build the case, with no need for new trials if trends hold steady. Key breakthroughs include the FDA’s reversal to accept the more sensitive cUHDRS as an endpoint—a victory families and researchers have fought for over seven years—and recognition of NFL as a marker for tracking the impact of disease-modifying therapies. These milestones could dramatically speed up approvals for treatments across the board.

A message from uniQure,

We are deeply appreciative of the support that we’ve received from the community and for the pioneering individuals who have volunteered to be a part of the study. Only through such collaborations can we continue our mission to develop treatments with the aim of modifying the course of Huntington’s disease. We look forward to continuing to work with you and the patient community as the AMT-130 program advances.

Read more about what this means and what is AMT- 130 in this HD Buzz article

You can read the full press release on the uniQure website

Glossary

What is cUHDRS?

cUHDRS, or the Composite Unified Huntington’s Disease Rating Scale. The cUHDRS is a sensitive way to measure clinical progression of HD. Along with the ability to determine how well someone functions day-to-day, the cUHDRS also includes tests that assess movement control, capacity to pay attention, and memory.

What is NfL?

NfL is a molecule that’s released from brain cells as they’re damaged. We know that NfL levels increase as HD progresses. So lots of researchers think that if we can show that a medication can hold NfL levels steady or make them decrease, that suggests improved brain cell health.

Read more about Gene Therapy.