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Data updated: Jun 28, 2026

PTC Therapeutics

Biotech
RespiratoryOncologyNeurology Execution: Fair

PTC Therapeutics is a biotechnology company focused on Respiratory, Oncology, Neurology.

2024
Since
1
Drugs
-
Trials
1
New Drugs (2yr)

PTC Therapeutics at a Glance

  • Fast trial execution (26 months median completion)

PTC Therapeutics's Key Drugs

PTC Therapeutics's core commercial portfolio centers on KEBILIDI, spanning Respiratory and Oncology — its most strategically important drugs approved in the last 15 years.

KEBILIDI
DDC Approved 2024

PTC Therapeutics's Recent FDA Approvals

PTC Therapeutics secured 1 new FDA approval for novel drugs (NDA/BLA) in the last two years.

KEBILIDI BLA
2024-11-12
eladocagene exuparvovec-tneq
1 INDICATIONS AND USAGE KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on the change from baseline in gross motor milestone achievement at 48 weeks post treatment [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. KEBILIDI is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on change from baseline in gross motor milestone achievement at 48 weeks post-treatment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. ( 1 , 14 )

PTC Therapeutics's Therapeutic Areas

PTC Therapeutics's approved drugs and pipeline span 4 therapeutic areas, led by Respiratory and Oncology. Weighted by commercial stage — approved drugs count most, then late-stage trials.

Respiratory 69%
0 drugs Phase 3: 5
Oncology 21%
0 drugs Phase 2: 1 Phase 1: 4
Neurology 7%
0 drugs Phase 2: 1
Gastroenterology 3%
0 drugs Phase 1: 1

PTC Therapeutics's Top Competitors

PTC Therapeutics's closest competitors by therapeutic-area and drug-target overlap include Bristol-Myers Squibb, EISAI INC, and GE HEALTHCARE. Ranked by overlap in therapeutic areas and drug targets across approved drugs and pipeline.

Bristol-Myers Squibb big-pharma
Oncology, Gastroenterology, Neurology
EISAI INC big-pharma
Oncology, Neurology, Gastroenterology, Respiratory
GE HEALTHCARE specialty
Neurology, Oncology, Respiratory
Pfizer big-pharma
Oncology, Neurology
AZURITY specialty
Oncology, Neurology, Respiratory

PTC Therapeutics Pipeline Snapshot

PTC Therapeutics has 12 active clinical programs from ClinicalTrials.gov — 5 Phase 3, 2 Phase 2 and 5 Phase 1.

5
Phase 3
2
Phase 2
5
Phase 1

Phase 3 Readout Calendar Pro

2 Phase 3 trials with confidence-graded primary completion dates.

Full calendar →
Q4 2027
Vatiquinone
Friedreich Ataxia
Estimated · aging NCT05515536
Q1 2026
Ataluren
Duchenne Muscular Dystrophy
Completed · awaiting NCT01247207

Anchored on CT.gov primary completion date. Topline announcements typically precede this by 3–9 months. Confidence labels combine date type (ACTUAL/ESTIMATED) with last-update freshness.

Pro Intelligence Preview

Deep insights for investors and analysts

Revenue Insights

  • • KEBILIDI leads revenue
  • • Drug-level revenue analysis

Trial Catalysts

  • • Respiratory pipeline focus
  • • 2 Phase 3 readouts tracked
View readout calendar →

Patent Risk

  • • Patent expiration timeline
  • • Revenue at risk analysis

Compare Companies

  • • Side-by-side pipeline analysis
  • • Revenue & market share comparison

Execution Intelligence

  • • Phase 3: 9/17 completed
  • • Speed: 26 months avg
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Revenue forecasts • Trial milestones • Patent risk modeling

How We Calculate These Metrics

Execution Quality Score

Measures trial completion rates weighted by phase importance. Phase 3 trials count 4x more than Phase 1 because late-stage completion is harder and more valuable.

What counts as "completed": A trial that reached its planned endpoint and reported results. Trials that were terminated early, withdrawn, or suspended are not counted as completed—these often indicate safety issues, lack of efficacy, or strategic pivots.

  • Excellent (80%+): Top-tier execution, most trials reach planned endpoints
  • Good (60-79%): Strong execution with occasional early terminations
  • Fair (40-59%): Average execution, notable rate of terminated/withdrawn trials
  • Needs Improvement (under 40%): High termination rates, execution challenges