Organization (Industry)
PTC THERAPEUTICS, INC.
ClinicalTrials.gov lists PTC THERAPEUTICS, INC. as lead sponsor of 65 trials. It has 25 active development programs and 5 approved assets.
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Trials started per year with this organization as sponsor or collaborator, 1990 onward. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 3 registered with a 2026 start date. The dashed outline is the pace from the 3 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 2.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Logo PTC Therapeutics, via Wikimedia Commons, Public domain. Shown to identify the organization; it may be a registered trademark.
This page covers PTC THERAPEUTICS, INC.: 65 lead-sponsored trials and 3 legal-entity registrations.
Disease areas (50)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Muscular Dystrophy, Duchenne | 18 |
| Mitochondrial Diseases | 11 |
| Cystic Fibrosis | 9 |
| Friedreich Ataxia | 6 |
| Leigh Disease | 5 |
Showing 5 of 50 disease areas.
Trials by start year as sponsor or collaborator (19)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2026 (partial year) | 3 |
| 2024 | 1 |
| 2022 | 6 |
| 2021 | 3 |
| 2020 | 3 |
| 2019 | 6 |
| 2018 | 5 |
| 2017 | 2 |
| 2016 | 6 |
| 2015 | 4 |
Showing 10 of 19 years.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (25)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| EPI-743 | Mitochondrial Diseases | Phase 3 | 8 |
| EPI-743 | Friedreich Ataxia | Phase 3 | 5 |
| PTC923 | Phenylketonurias | Phase 3 | 4 |
| Ataluren | Muscular Dystrophy, Duchenne | Phase 3 | 13 |
| EPI-743 | Leigh Disease | Phase 3 | 4 |
Showing 5 of 25 active programs.
Approved assets (5)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| Ataluren | 28 |
| Deflazacort | 8 |
| Dacarbazine | 2 |
| DOCETAXEL | 1 |
| RIFAMPIN | 1 |
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 63% hit target (n=30) | 37% on time (n=30) |
Journals published in (57)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Journal of comparative effectiveness research | 5 |
| PloS one | 4 |
| The Journal of clinical investigation | 4 |
| Journal of clinical oncology : official journal of the American Society of Clinical Oncology | 3 |
| Lancet (London, England) | 3 |
Showing 5 of 57 journals.
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 176 | 37 | 2025-12-30 |
SEC-reported annual financials (11)
Shown in this order: newest fiscal year first; SEC EDGAR XBRL companyfacts, USD as reported.
| Fiscal year | Revenue (USD) | Net income (USD) | R&D (USD) |
|---|---|---|---|
| 2025 | 1,730,655,000 | 682,644,000 | 455,249,000 |
| 2024 | 806,780,000 | -363,295,000 | 534,480,000 |
| 2023 | 937,822,000 | -626,604,000 | 666,563,000 |
| 2022 | 698,801,000 | -559,017,000 | 651,496,000 |
| 2021 | 538,593,000 | -523,901,000 | 540,684,000 |
| 2020 | 380,766,000 | -438,160,000 | 477,643,000 |
| 2019 | 306,980,000 | -251,576,000 | 257,452,000 |
| 2018 | 264,734,000 | -128,081,000 | 171,984,000 |
| 2017 | 194,392,000 | -79,000,000 | 117,456,000 |
| 2016 | 82,705,000 | -142,110,000 | 117,633,000 |
Showing 10 of 11 fiscal years.
Financing and grant events (37)
Shown in this order: most recent first.
| Date | Round type | Amount (USD) | Source |
|---|---|---|---|
| 2026-06-18 | Debt financing | 550,000,000 | Sec extracted |
| 2026-06-18 | Debt financing | not disclosed | Sec extracted |
| 2026-06-18 | Debt financing | not disclosed | Sec extracted |
| 2026-06-15 | Debt financing | not disclosed | Sec extracted |
| 2025-12-29 | Royalty / licensing | 240 | Sec extracted |
Showing 5 of 37 events.
FDA inspection record (1)
| Latest classification | Inspections |
|---|---|
| NAI | 4 |
Climate targets, as filed (1)
| Reporting year | Net-zero target |
|---|---|
| 2025 | not disclosed |
Workforce and board diversity, as filed (1)
| Reporting year | Workforce % women | Board % women |
|---|---|---|
| 2025 | not disclosed | not disclosed |
Organization relationships (19)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Genzyme, a Sanofi Company | Co sponsors trial with | 5 |
| Cystic Fibrosis Foundation | Co sponsors trial with | 4 |
| Government of the United States of America | Co sponsors trial with | 3 |
| Roche (Switzerland) | Co sponsors trial with | 1 |
| Medical University of South Carolina | Co sponsors trial with | 1 |
Showing 5 of 19 relationships.
Latest dated events (292)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-08-31 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-08-15 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-06-18 | Debt facility | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2026-06-18 | Debt facility | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2026-06-18 | Debt facility | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
Showing 5 of 292 events.
Biomarker capability evidence (276)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ALB | Trial sponsor | Named in trial inclusion criteria |
| ALB | Trial collaborator | Named in trial inclusion criteria |
| ALB | Trial sponsor | Primary endpoint in a Phase 3 trial |
| CDKL5 | Trial collaborator | Named in trial inclusion criteria |
| CFTR | Trial sponsor | Named in trial inclusion criteria |
Showing 5 of 276 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, fda warning letters, fda untitled letters, fda recalls, fda import refusals, manufacturing and distribution sites, clia clinical laboratory certificates, and biomarkers this organization publishes on.