Headquartered in Warren, NJ Fiscal year ends December NASDAQ: PTCT ptcbio.com
PTC Therapeutics is a global biopharmaceutical company that discovers, develops and commercializes medicines for children and adults with rare disorders.
**Commercial products and royalties:**
- Sephience (sepiapterin) for phenylketonuria, now its largest product, launched in the US and expanding internationally.
- Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy.
- Emflaza (deflazacort) for Duchenne muscular dystrophy.
- Upstaza/Kebilidi gene therapy for AADC deficiency.
- Tegsedi and Waylivra.
- Royalty revenue on Roche's Evrysdi (risdiplam) for spinal muscular atrophy, under an SMA collaboration; part of these future royalties has been sold.
**Pipeline:**
- ST-920, a one-time AAV gene therapy for Fabry disease acquired from Sangamo Therapeutics, with a BLA submission planned for Q4 2026.
- Votoplam for Huntington's disease, partnered with Novartis.
- Vatiquinone for Friedreich's ataxia.
- Earlier-stage candidates PTC844 (DHODH inhibitor), PTC612 (oral NLRP3 inhibitor) and PTC303 (MSH3 splicing).
The company draws on its splicing and small-molecule science and a global commercial infrastructure. Updated
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Lee Jackson PTC Therapeutics is a global biopharmaceutical company that discovers, develops and commercializes medicines for children and adults with rare disorders. **Commercial products and royalties:** - Sephience (sepiapterin) for phenylketonuria, now its largest product, launched in the US and expanding internationally.…
The Wall Street consensus 12-month price target for PTCT is $100.47.
