UniQure submits Huntington's gene therapy to FDA after agency reversal
UniQure has submitted its experimental Huntington's gene therapy to the Food and Drug Administration for accelerated approval review, the company announced. The move follows a reversal by the FDA in June. Agency officials who have since left had earlier told the company it could not submit the therapy based on its Phase 1/2 trial. That trial showed the medicine could slow progression of the fatal neurodegenerative disease. UniQure said it had also applied for approval to the U.K.'s regulatory agency.
Huntington's patients have no disease-modifying treatments available.
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