S&P 500 slips on report Fed’s Waller leading race to replace Powell; tech shines
Investing.com -- Shares of uniQure N.V. (NASDAQ:QURE) surged 39% following the announcement that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to AMT-130, its gene therapy candidate for the treatment of Huntington’s disease. This significant regulatory milestone, which aims to expedite the development and review process of AMT-130, has been well-received by investors, signaling potential for an accelerated path to market for the treatment.
The FDA’s Breakthrough Therapy designation is supported by clinical data from ongoing Phase I/II trials, which have indicated that AMT-130 could slow disease progression. The optimism surrounding uniQure’s stock is further bolstered by a comprehensive set of FDA designations for AMT-130, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations, reflecting the drug’s potential to address the urgent need for effective treatments for Huntington’s disease.
uniQure’s Chief Medical (TASE:BLWV) Officer, Walid Abi-Saab, M.D., highlighted the significance of the designation, stating, “Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression.” He expressed gratitude for the FDA’s commitment to advancing gene therapies for patients with critical unmet needs and conveyed the company’s eagerness to work with the agency to deliver AMT-130 to patients as swiftly as possible.
Huntington’s disease is a debilitating neurodegenerative disorder characterized by motor symptoms, behavioral abnormalities, and cognitive decline, resulting in progressive deterioration. The disease affects approximately 70,000 people in the U.S. and Europe, with no current disease-modifying therapies available. The Breakthrough Therapy designation is particularly significant for uniQure, as it indicates that AMT-130 may offer substantial improvement over existing therapies for a serious condition, based on preliminary clinical evidence.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.