PTC Therapeutics announced on September 26, 2024, that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to its PTC518 program for the treatment of Huntington's disease. This designation is awarded to promising therapies for serious or life-threatening conditions with unmet medical needs, facilitating development and expediting the review process.
PTC518 is an oral small molecule splicing agent designed to reduce the production of the mutated Huntingtin protein. Interim 12-month data from the PIVOT-HD study, reported in June 2024, demonstrated dose-dependent lowering of mutant HTT protein in both blood cells and cerebrospinal fluid.
The study also showed early signals of dose-dependent favorable clinical effect on key disease measurements, including the total motor score and the cUHDRS scale. Importantly, PTC518 maintained a favorable safety and tolerability profile with no treatment-related NfL spikes.
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