UniQure submits experimental Huntington's gene therapy to FDA for accelerated review

UniQure announced that it has filed its experimental gene therapy for Huntington's disease with the U.S. Food and Drug Administration, seeking accelerated approval.

The submission follows earlier guidance from the agency that had questioned the company’s ability to file the therapy, prompting UniQure to address those concerns before moving forward.

The FDA will now evaluate the data under the accelerated pathway, which could shorten the time to market if the therapy demonstrates significant benefit.

In related news, recent White House agreements on drug pricing target Medicaid discounts but do not yet affect Medicare, limiting their immediate impact on overall drug costs.