PTC Therapeutics
Quick facts
Phase 3 pipeline
- Ataluren (PTC124®) · Rare genetic diseases
Ataluren allows ribosomes to read through premature stop codons (nonsense mutations) and produce full-length, functional proteins. - PTC923 · Rare genetic/metabolic disorders
PTC923 is a small-molecule inhibitor of pyruvate dehydrogenase kinase (PDK) that restores pyruvate dehydrogenase (PDH) activity to improve mitochondrial energy metabolism.
Phase 2 pipeline
- CNSA-001 · Rare Diseases
CNSA-001 is a small molecule that targets the underlying cause of a specific disease. - PTC124
- PTC518
Phase 1 pipeline
- Deflazacort and Clarithromycin
- Deflazacort and rifampin
Deflazacort is a corticosteroid with anti-inflammatory effects; rifampin induces CYP3A4 enzymes that metabolize deflazacort. - PTC596
Competitive intelligence
For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:
Frequently asked questions about PTC Therapeutics
What is PTC Therapeutics's pipeline?
PTC Therapeutics has 2 drugs in Phase 3, 3 in Phase 2, 3 in Phase 1. Late-stage candidates include Ataluren (PTC124®), PTC923.
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