Company intelligence / Profile preview
PTC Therapeutics, Inc.
South Plainfield, New Jersey, United Statesptcbio.com ↗
- Ownership
- Public · PTCT
- Employees
- ~5000
- Development stage
- Commercial
01
Overview
PTC Therapeutics has multiple approved products including Translarna for nonsense mutation Duchenne muscular dystrophy, Emflaza for Duchenne muscular dystrophy, Kebilidi/Upstaza (gene therapy) for AADC deficiency, and Evrysdi for spinal muscular atrophy. The company recently received a positive CHMP opinion for Sephience (sepiapterin) for PKU treatment and submitted a new drug application for vatiquinone to treat Friedreich ataxia.
- Therapeutic areas
- OncologyRare DiseasesNeurologyMetabolic Disorders
- Modalities
- Small moleculesGene therapySplicing modifiers
- Industry
- Biotech
02
Drug pipeline
22 assets21 more assets in the full pipeline.
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Partnerships
RocheNovartis Pharmaceuticals Corporation
04
Financials
- Market cap
- $5.6B
- Total revenue
- $360.5M
- Enterprise value
- $4.1B
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