FDA Fast Tracks PTC518 for Huntington Disease

The Food and Drug Administration (FDA) has granted Fast Track designation to PTC518 for the treatment of Huntington disease (HD).

PTC518, an orally taken small molecule discovered from RNA splicing technology, decreased production of the Huntingtin protein mutation, which is known to lead to neuron injury and death. The Company claim the investigational treatment is able to penetrate the blood brain barrier, is titratable, selective and not effluxed.

The designation is supported by positive interim results from the 12-month PIVOT-HD study (ClinicalTrials.gov Identifier: NCT05358717), an interim data of the phase 2, randomized, placebo-controlled, dose-ranging study evaluating the safety and efficacy of PTC518 vs placebo in patients with HD. 

Data at 12 months showed that those treated with PTC518 10mg had a 43% lowering of the mutant Huntingtin protein in blood cells. Similar results were seen for dose-dependent lowering of mutant Huntingtin protein in the cerebrospinal fluid. There were also positive dose-dependent effects on important disease measurements such as total motor score and the Composite Unified Huntington Disease Rating Scale (cUHDRS).

PTC518 based on interim data was seen to be safe and well tolerated with no increases in treatment-related neurofilament light (NfL).

“The granting of Fast Track designation to the PTC518 program further supports the potential of PTC518 to provide a disease modifying therapy to Huntington’s disease patients,” said Matthew B. Klein, MD, Chief Executive Officer of PTC Therapeutics. “We look forward to working collaboratively with FDA to continue to advance the program as efficiently as possible.”

This article originally appeared on MPR