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Data updated: Jun 28, 2026

PTC THERAP

Pharma
RespiratoryOncologyNeurology Execution: Fair

PTC THERAP is a pharmaceutical company focused on Respiratory, Oncology, Neurology. Key products include SEPHIENCE.

2017
Since
2
Drugs
-
Trials
1
New Drugs (2yr)
Modality:
2 Small Molecules

FDA Novel Drug Designations

(2017-2025)
2 Novel 1 First-in-Class 2 Orphan 1 Priority Review 1 Fast Track
See 2 novel approvals →
2025 SEPHIENCE (SEPIAPTERIN) Orphan
2017 EMFLAZA (DEFLAZACORT) First-in-Class · Orphan · Priority Review · Fast Track

PTC THERAP at a Glance

  • Fast trial execution (26 months median completion)

PTC THERAP's Key Drugs

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

SEPHIENCE
Rare Disease

Hyperphenylalaninemia

PAH Approved 2025
EMFLAZA
Rare Disease

Duchenne Muscular Dystrophy

Glucocorticoid receptor Approved 2017

PTC THERAP's Recent FDA Approvals

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

SEPHIENCE NDA
2025-07-28
sepiapterin
Hyperphenylalaninemia

PTC THERAP's Therapeutic Areas

PTC THERAP's approved drugs and pipeline span 4 therapeutic areas, led by Respiratory and Oncology, across 0 biologic and 2 small-molecule drugs. 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 THERAP's Top Competitors

PTC THERAP'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 THERAP Pipeline Snapshot

PTC THERAP 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

Pro Intelligence Preview

Deep insights for investors and analysts

Revenue Insights

  • • SEPHIENCE leads revenue
  • • 2 key drugs tracked

Trial Catalysts

  • • Respiratory pipeline focus
  • • Phase 3 readout tracking

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
Unlock Full Intelligence

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