PTC Therapeutics is a U.S. pharmaceutical company focused on small-molecule and gene-therapy approaches that modulate post-transcriptional control mechanisms in rare diseases. The company is headquartered in Warren Township, New Jersey and was founded in 1998. It is publicly traded on Nasdaq under PTCT.
The Consolidated Appropriations Act of 2026 extends the FDA's authority to award rare pediatric disease priority review vouchers through September 30, 2029, restoring a key incentive for developing therapies for rare pediatric diseases.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following FDA feedback that the data are unlikely to meet the threshold for substantial evidence of effectiveness.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07159139 |
Vatiquinone Expanded Access Protocol |
NO_LONGER_AVAILABLE | |
| NCT06302348 |
A Study of Sepiapterin in Participants With Phenylketonuria (PKU) |
RECRUITING | PHASE3 |
| NCT05515536 |
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05485987 |
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia |
COMPLETED | PHASE2 |
| NCT05358717 |
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) |
COMPLETED | PHASE2 |
| NCT05349721 |
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS |
TERMINATED | PHASE2 |
| NCT05269355 |
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS) |
TERMINATED | PHASE2/PHASE3 |
| NCT05218655 |
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease |
COMPLETED | PHASE3 |
| NCT05166161 |
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05099640 |
A Study of PTC923 in Participants With Phenylketonuria |
COMPLETED | PHASE3 |
