Organization (Industry)
AOP Orphan Pharmaceuticals GmbH
ClinicalTrials.gov lists AOP Orphan Pharmaceuticals GmbH as lead sponsor of 16 trials. It has 5 active development programs and 10 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: 1 registered with a 2026 start date. The dashed outline is the pace from the 1 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 1.0 trial starts a year over the five years to September, under the 5 a year the method needs.
This page covers AOP Orphan Pharmaceuticals GmbH: 16 lead-sponsored trials and 2 legal-entity registrations.
Disease areas (9)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Hematologic Neoplasms | 5 |
| Polycythemia Vera | 5 |
| Hypertension, Pulmonary | 4 |
| Pulmonary Arterial Hypertension | 4 |
| Thrombocythemia, Essential | 4 |
Showing 5 of 9 disease areas.
Trials by start year as sponsor or collaborator (12)
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) | 1 |
| 2024 | 2 |
| 2023 | 2 |
| 2017 | 2 |
| 2015 | 1 |
| 2014 | 2 |
| 2013 | 2 |
| 2012 | 2 |
| 2011 | 1 |
| 2010 | 2 |
Showing 10 of 12 years.
Recruitment duration, a model prediction: 1 trial 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 (5)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| preservative-free parenteral treprostinil | Pulmonary Arterial Hypertension | Phase 3 | 1 |
| preservative-free parenteral treprostinil | Hypertension, Pulmonary | Phase 3 | 1 |
| Ropeginterferon Alfa-2B | Thrombocythemia, Essential | Phase 3 | 1 |
| Treprostinil | Pulmonary Arterial Hypertension | Not reported | 1 |
| Treprostinil | Hypertension, Pulmonary | Not reported | 1 |
Approved assets (10)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| Generic treprostinil sodium + Standard of Care (Double Oral) | 1 |
| Standard of Care - Double Oral | 1 |
| HYDROXYUREA | 2 |
| Thromboreductin | 1 |
| Ropeginterferon Alfa-2B | 1 |
Showing 5 of 10 approved assets.
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 |
|---|---|
| not enough data | 0 of 1 on time (n=1) |
Journals published in (35)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Blood | 28 |
| Leukemia | 11 |
| Journal of clinical oncology : official journal of the American Society of Clinical Oncology | 8 |
| The New England journal of medicine | 7 |
| The Journal of clinical investigation | 3 |
Showing 5 of 35 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 |
|---|---|---|
| 7 | 2 | 2024-04-23 |
FDA import refusals (1)
| Refused import lines | Latest refusal |
|---|---|
| 1 | 2021-06-30 |
Manufacturing and distribution sites (2)
Shown in this order: severity rank then name.
| Facility | Location | Regulators | Member organization |
|---|---|---|---|
| Aop Orphan Pharmaceuticals GmbH | Vienna, AT | EMA, Health Canada | |
| AOP Orphan Pharmaceuticals AG | Wien, AT | EMA |
Organization relationships (4)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| PharmaLex Belgium | Co sponsors trial with | 1 |
| Aixial s.r.o. | Co sponsors trial with | 1 |
| ANOVA CRO s.r.o. | Co sponsors trial with | 1 |
| GCP-Service International Ltd. & Co. KG | Co sponsors trial with | 1 |
Latest dated events (27)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-07 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2024-07-31 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2024-07-31 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2024-04-23 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2023-12-06 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
Showing 5 of 27 events.
Biomarker capability evidence (50)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ABL1 | Trial collaborator | Named in trial inclusion criteria |
| ABL1 | Trial collaborator | Named in trial inclusion criteria |
| ABL1 | Trial collaborator | Primary endpoint in a Phase 3 trial |
| ALB | Trial sponsor | Named in trial inclusion criteria |
| BCR | Trial collaborator | Named in trial inclusion criteria |
Showing 5 of 50 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, sec-reported annual financials, financing and grant events, fda warning letters, fda untitled letters, fda inspection record, fda recalls, climate targets, as filed, workforce and board diversity, as filed, clia clinical laboratory certificates, and biomarkers this organization publishes on.