PTCT (PTC Therapeutics Inc.) stock: $65.56, $5.47B market cap, $2.23B cash, $3.78B EV, $5.87B fully diluted market cap. Next expected readout: Sep-27. Lead program: Vatiquinone (Friedreich Ataxia), Phase 3. Data from Fully Diluted's biotech stocks database.
Pipeline assets
Sephience
Approved · Active
Aliases sepiapterin
Target phenylalanine hydroxylase (PAH)
Indications- Phenylketonuria (PKU) (Approved)
Status note Approved EC Jun 2025 (EEA), FDA Jul 2025 (US, age 1 month+), Japan Dec 2025, Brazil Feb 2026, plus additional geographies.
Translarna
Stage not disclosed · Active
Aliases ataluren
Indications- Nonsense mutation Duchenne muscular dystrophy (nmDMD) (Stage not disclosed)
Status note EC adopted CHMP negative opinion Mar 2025 (conditional MA not renewed; continued use possible via EU Directive 2001/83 Articles 117(3)/5(1)). US NDA resubmission withdrawn Feb 2026 after FDA feedback; no further US development planned. Authorizations remain in some geographies outside the EEA.
Emflaza
Approved · Active
Aliases deflazacort
Indications- Duchenne muscular dystrophy (Approved)
Status note Approved in US for DMD patients 2 years and older; lost US market exclusivity under the Orphan Drug Act.
Upstaza
Approved · Active Modality: gene therapy
Aliases eladocagene exuparvovec, Kebilidi
Target AADC (dopa decarboxylase gene)
Indications- Aromatic L-amino acid decarboxylase (AADC) deficiency (Approved)
Status note Approved EC Jul 2022 (EEA, 18 months+), UK Nov 2022, FDA Nov 2024 (marketed as Kebilidi in the US).
Evrysdi
Approved · Active
Aliases risdiplam
Target SMN2 (splicing modifier)
Partnerships Roche + SMA Foundation: SMA License Agreement (2011); PTC receives milestones and royalties.
Indications- Spinal muscular atrophy (SMA) (Approved)
Status note Approved FDA Aug 2020, EC Mar 2021, 100+ countries; label expanded to infants under 2 months (FDA May 2022, EC Aug 2023).
Votoplam
Phase 3 · Active Modality: splicing modifier
Aliases PTC518
Target huntingtin (HTT)
Partnerships Novartis: License and Collaboration Agreement (closed Jan 2025, $1.0B upfront); $50M milestone on Phase 3 initiation; development/regulatory/sales milestones + profit sharing + royalties.
Indications- Huntington's disease (Phase 3)
Status note HD splicing-platform program. Novartis license closed Jan 2025 ($1.0B upfront). Global Phase 3 commenced Apr 2026 (~770 early symptomatic patients; interim analysis planned).
Vatiquinone
Stage not disclosed · Active Modality: small molecule
Target 15-lipoxygenase
Indications- Friedreich's ataxia (Stage not disclosed)
Status note Registration-directed Phase 3 MOVE-FA did not meet its primary endpoint (May 2023). NDA submitted Dec 2024, accepted Feb 2025, CRL Aug 2025. FDA suggested additional study; PROVE-FA open-label study (~120 patients, ages 7-21) planned Q3 2026.
PTC612
Phase 1 · Active Modality: small molecule (oral)
Target NLRP3
Status note Oral NLRP3 inhibitor; Phase 1 in healthy volunteers initiated Q2 2026.
PTC844
Stage not disclosed · Active Modality: small molecule (oral)
Target DHODH
Indications- Rheumatoid Arthritis (Phase 2)
Status note Next-generation DHODH inhibitor; Phase 2a planned.
Tegsedi
Approved · Active
Aliases inotersen
Target TTR
Partnerships Ionis/Akcea: PTC holds commercialization rights in Latin America and the Caribbean.
Indications- Hereditary transthyretin amyloidosis polyneuropathy (Approved)
Status note Approved US, EU, Brazil for stage 1/2 polyneuropathy in hATTR amyloidosis.
Waylivra
Approved · Active
Aliases volanesorsen
Partnerships Ionis/Akcea: PTC holds commercialization rights in Latin America and the Caribbean.
Indications- Familial chylomicronemia syndrome (Approved)
- Familial partial lipodystrophy (Approved)
Status note Approved Brazil (Aug 2021, first FCS treatment; Dec 2022 for familial partial lipodystrophy) and EU for FCS.
Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.