Weekly Rundown: uniQure will seek approval for Huntington's disease gene therapy after switchup at FDA

Weekly Rundown: uniQure will seek approval for Huntington's disease gene therapy after switchup at FDA Gene therapies dominated the news this week, with positive results for both a CRISPR and non-viral therapy, and a U-turn decision from the FDA. Written byDDN editorial team https://www.drugdiscoverynews.com/weekly-rundown-uniqure-will-seek-approval-for-huntington-s-disease-gene-therapy-after-switchup-at-fda-17271 uniQure will seek approval for Huntington’s disease gene therapy after switchup at FDA uniQure announced that they will move forward with submitting a Biologics License Application (BLA) to the FDA for their gene therapy to treat Huntington’s disease. The news follows the FDA’s prior rejection of their data package back in March, which a former top FDA official called “truly evil.” The decision was part of a string of rejections under former FDA Director of the Center for Biologics Evaluation and Research, Vinay Prasad. After Prasad left the FDA for a second time in April and FDA Commissioner Martin Makary’s exit in May, FDA officials met with uniQure and said that they will now accept a three-year analysis from their Phase 1/2 trial as the primary basis for their BLA. That trial showed that their one-time gene therapy led to a 75 percent slowing of disease at 36 months at the higher dose. UniQure’s stock rose 70 percent after the announcement in anticipation of the possible upcoming FDA approval. “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,” said Matt Kapusta, CEO at uniQure. – Allison Whitten