UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency

Thomas Fuller | SOPA Images | Lightrocket | Getty Images UniQure plans to seek FDA approval of its experimental gene therapy for Huntington's disease, the company said Wednesday, months after previous agency leaders criticized the evidence backing the application. UniQure said the FDA in a recent meeting communicated that a three-year analysis from a Phase 1/2 study would support an accelerated approval of UniQure's gene therapy for Huntington's, a rare hereditary disease that gradually destroys nerve cells in the brain. As a result of the meeting, UniQure plans to submit its application to the FDA in the third quarter of this year. Shares of UniQure soared 70% on Wednesday. The new FDA guidance represents a stunning reversal from March, when the regulator told Uniqure that its clinical trial data wouldn't support an application and publicly criticized the company. UniQure became a prime example in a series of reversals where companies said the FDA had changed its previous guidance, hitting rare disease drugmakers especially hard. Many of those decisions happened under former FDA Commissioner Marty Makary, who left the agency in May. …