Sept 2 (Reuters) – Dutch gene therapy developer uniQure said on Wednesday it had applied for U.S. approval of its experimental treatment for Huntington’s disease after the FDA reversed its earlier position on the drug, sending the company’s shares up 5% in premarket trading.
Huntington’s disease is a rare inherited brain disorder that causes movement difficulties and triggers behavioural changes and cognitive decline. No approved drugs currently exist that help slow progression.
Here are more details:
• The company said it has submitted a marketing application to the U.S. Food and Drug Administration for accelerated approval of ifezuntirgene inilparvovec or AMT-130.
• uniQure has asked the FDA for priority review, which, if granted, would shorten the agency’s review period to six months from the standard 10 months.
• The development follows months of friction between uniQure and the FDA, which had earlier indicated that existing data was insufficient to support a marketing application, with a senior FDA official calling uniQure’s experimental gene therapy a “failed product”.
• In June, uniQure said the regulator reversed its earlier position and said it would accept existing trial data for an accelerated review .
• uniQure’s application is backed by three-year data showing the therapy slowed disease progression versus comparable patients in an external Huntington’s disease database. The company expects to present four-year data later this quarter.
• The FDA faced criticism for seeking a new trial that included a placebo-surgery control group, requiring some patients to undergo invasive brain procedures without receiving the gene therapy.
• The dispute played out during the tenure of Vinay Prasad, then the FDA’s top regulator overseeing vaccines and gene therapies. Prasad, who was criticized for a series of decisions affecting rare-disease drug developers, left the agency in April.
• The FDA’s acting commissioner, Kyle Diamantas, who took over in May, has since restored hope among rare disease groups as he looks to repair relationships with several biotechs following Marty Makary’s turbulent tenure as the chief.
• uniQure also filed an application for the therapy with Britain’s medicines regulator.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Diti Pujara)
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