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Search results for "EU ORPHAN DRUG" in comments (approximate match)
Status:
Investigational
Source:
NCT03061812: Phase 3 Interventional Completed Small Cell Lung Cancer
(2017)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT01963598: Phase 2 Interventional Completed Sarcopenia
(2013)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT04050462: Phase 2 Interventional Active, not recruiting Hepatocellular Carcinoma
(2019)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT04535674: Phase 2 Interventional Completed COVID-19 Induced Pneumonia
(2020)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT04587830: Phase 2 Interventional Recruiting Glioblastoma Multiforme (GBM)
(2020)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT02573467: Phase 3 Interventional Completed Sporadic Inclusion Body Myositis
(2015)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT01098266: Phase 3 Interventional Completed Malignant Pleural Mesothelioma
(2010)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT04176133: Phase 2 Interventional Completed Healthy
(2019)
Source URL:
Class:
PROTEIN
Status:
Investigational
Source:
NCT02095626: Phase 2 Interventional Completed PGD Primary Graft Dysfunction
(2013)
Source URL:
Class:
PROTEIN
Apeptico is developing solnatide (AP301), a dry powder inhalation and an inhaled aerosol formulation of a synthetic peptide for the treatment of acute lung injury, acute respiratory distress syndrome and pulmonary oedema caused by lung infection or high altitude. The AP301 peptide mimics the lectin-like domain of TNF-α. The synthetic peptide AP301 is composed of 17 amino acids and contains an intramolecular disulfide bond between the N-terminal and the C-terminal cysteine. AP301 interacts with the endothelial sodium channel (ENaC) and activates pulmonary liquid clearance both in vitro and in animal studies. The specific interaction of AP301 with both endogenously and heterologously expressed ENaC requires precedent binding to glycosylated extracellular loop(s). Solnatide has been granted Orphan Drug Designation for “Treatment of Primary Graft Dysfunction following Lung Transplantation” and for “Treatment of Pseudohypoaldosteronism Type 1B” by the Food and Drug Administration.
Status:
Investigational
Source:
INN:tiprelestat [INN]
Source URL:
Class:
PROTEIN
Tiprelestat (Elafin) is a potent Human neutrophil elastase inhibitor. Elafin is identical to the human protein elafin with high specificity for tissue destroying and inflammation promoting proteases. The development program of Elafin is focused on the late stage development of Elafin in major surgery and early stage development in pulmonary arterial hypertension (PAH). Elafin has received orphan drug designations in the USA and the EU for esophageal cancer surgery (ECS) and PAH. Tiprelestat is in phase II clinical trials for the treatment of myocardial reperfusion injury, postoperative inflammation and in phase I clinical trial for the treatment of pulmonary arterial hypertension.