FDA Grants Fast Track Designation to PTC518 Huntington’s Disease Program

PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression.   Programs with Fast Track designation can benefit from early interactions with the FDA, and may be eligible for priority review and accelerated approval. This follows the release of 12 month interim data from PTC’s Phase 2 study, which showed that participants receiving PTC518 had dose dependent lowering up to 43% of mutant Huntingtin protein in the blood. 

 “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, M.D., Chief Executive Officer of PTC Therapeutics. 

Read the press release here.