PTC Therapeutics Says FDA Grants Fast Track Designation For PTC518 To Treat Huntington's Disease

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Thursday that the FDA has granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. Fast Track designation is awarded to promising therapies for diseases of high unmet need.

The company said 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.

PTC518 was discovered using PTC's innovative splicing platform, following the successful discovery and development of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy.

PTC518, a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression. The orally bioavailable small molecule penetrates the blood brain barrier, is selective, titratable, and not effluxed.