Drug intelligence / Profile preview

molgramostim

Development stage
Unknown
Lead developer
Savara
Modality
Cytokines & Interferons → Recombinant Proteins and Enzymes
Administration
Inhalation
01

Overview

Molgramostim is a non-glycosylated, recombinant form of human granulocyte-macrophage colony-stimulating factor (GM-CSF), developed as an inhaled biologic therapy. It acts by stimulating the growth, differentiation, and function of myeloid lineage cells including granulocytes and macrophages. Its primary mechanism involves activating alveolar macrophages to restore surfactant clearance in the lungs, which is particularly relevant for diseases like autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim has been granted Orphan Drug Designation for aPAP by both FDA and EMA and is under development primarily as an inhaled solution. The drug has also been investigated in other rare lung conditions such as acute respiratory distress syndrome (ARDS), nontuberculous mycobacterial infections, cystic fibrosis, bronchiectasis, and COVID-19 pneumonia[1][2][3][4][5][6][7].

Brand names
MOLBREEVIMolgradex
Other names
granulocyte macrophage-colony stimulating factorrecombinant human granulocyte-macrophage colony-stimulating factorrhGM-CSF
02

Targets

CSF2R (Granulocyte-macrophage colony-stimulating factor receptor)

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