Overview
Molgramostim has been used in trials studying the treatment of Bronchiectasis, Cystic Fibrosis, Pulmonary Alveolar Proteinosis, Acute Respiratory Distress Syndrome, and Autoimmune Pulmonary Alveolar Proteinosis.
Indication
No indication information available.
Associated Conditions
No associated conditions information available.
Research Report
Molgramostim (DB12525): A Comprehensive Monograph on a Novel Inhaled Therapy for Autoimmune Pulmonary Alveolar Proteinosis
Executive Summary
Molgramostim is an investigational biotech therapeutic poised to address a significant unmet medical need in the treatment of autoimmune Pulmonary Alveolar Proteinosis (aPAP), a rare and debilitating lung disease. Developed by Savara Pharmaceuticals, Molgramostim is a recombinant, non-glycosylated form of human granulocyte-macrophage colony-stimulating factor (GM-CSF) formulated as an inhaled solution. Its mechanism of action directly targets the underlying pathophysiology of aPAP by restoring the function of alveolar macrophages, which are impaired by autoantibodies against endogenous GM-CSF. This targeted replacement therapy reactivates the natural clearance of lung surfactant, thereby improving pulmonary gas exchange.
The clinical development program has culminated in the pivotal Phase 3 IMPALA-2 trial, which demonstrated compelling and statistically significant efficacy across multiple domains. Treatment with inhaled Molgramostim resulted in marked improvements in pulmonary gas transfer, a key physiological measure of lung function. These objective gains were complemented by clinically meaningful enhancements in respiratory health-related quality of life and functional exercise capacity, directly linking the drug's physiological effect to tangible patient benefits. Furthermore, the therapy reduced the underlying surfactant burden in the lungs and decreased the need for whole lung lavage, the current invasive standard of care.
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
---|---|---|---|---|---|
2024/08/09 | N/A | AVAILABLE | |||
2024/05/29 | Phase 3 | Not yet recruiting | |||
2023/03/16 | Phase 2 | Recruiting | |||
2022/08/15 | Phase 1 | Not yet recruiting | Second Affiliated Hospital of Soochow University | ||
2021/12/20 | Phase 2 | Terminated | |||
2020/09/30 | Phase 2 | Completed | |||
2020/09/10 | Phase 3 | Active, not recruiting | |||
2018/07/24 | Phase 2 | Terminated | |||
2018/03/29 | Phase 3 | Terminated | |||
2018/02/05 | Phase 2 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
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No FDA approvals found for this drug. |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
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No EMA approvals found for this drug. |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
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No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
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No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
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No PPB approvals found for this drug. |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
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No TGA approvals found for this drug. |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
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No Health Canada approvals found for this drug. |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
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No CIMA AEMPS (Spain) approvals found for this drug. |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Philippines FDA approvals found for this drug. |
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Saudi SFDA approvals found for this drug. |
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
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No Malaysia NPRA approvals found for this drug. |
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
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No UK EMC drug information found for this drug. |
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