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Molgramostim

Generic Name
Molgramostim
Drug Type
Biotech
CAS Number
99283-10-0
Unique Ingredient Identifier
B321AL142J

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

Published: Sep 26, 2025

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.

Continue reading the full research report

FDA Drug Approvals

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No FDA approvals found for this drug.

EMA Drug Approvals

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HSA Drug Approvals

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No HSA approvals found for this drug.

NMPA Drug Approvals

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No NMPA approvals found for this drug.

PPB Drug Approvals

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No PPB approvals found for this drug.

TGA Drug Approvals

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No TGA approvals found for this drug.

Health Canada Drug Approvals

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No Health Canada approvals found for this drug.

CIMA AEMPS Drug Approvals

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No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

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No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

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No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

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No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

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No UK EMC drug information found for this drug.

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