NONE — The Food and Drug Administration reversed its previous position regarding an experimental gene therapy for Huntington's disease. The agency now finds a three-year analysis of a Phase 1/2 study that demonstrates patient benefit to be acceptable in support of a marketing application, according to a recent meeting with the FDA.

UniQure, the manufacturer of the experimental therapy AMT-130, plans to submit a marketing application for accelerated approval during the third quarter of this year. This decision follows the FDA informing the company in March that its clinical trial data at that time would not support a marketing application.

A senior FDA official had previously requested the company conduct a placebo-controlled trial to demonstrate the therapy's efficacy. The company stated that it would be unethical to require a control group to undergo a sham surgical procedure. The company compared disease progression in treated patients against typical disease progression using an external database. In a Phase 1/2 clinical trial, AMT-130 slowed disease progression by 75 percent.

AMT-130 is administered through a surgical procedure that delivers the therapy directly into the brain over several hours. Huntington's disease is a rare hereditary condition that progressively destroys nerve cells in the brain. Accelerated approval allows a treatment to enter the market provided the sponsor demonstrates benefit in a subsequent confirmatory study. The FDA requested that the company design a follow-up study comparing the therapy to the standard of care prior to submission. The company plans to finalize this study's design before submitting its marketing application. The company's stock increased by 70 percent on Wednesday.