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Molgramostim Open-label
Molgramostim Open-label is a Cytokine; GM-CSF receptor agonist Small molecule drug developed by Savara Inc.. It is currently in Phase 3 development for Pulmonary alveolar proteinosis (PAP). Also known as: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).
Molgramostim is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) that stimulates the production and activation of granulocytes and macrophages to enhance immune function.
Molgramostim is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) that stimulates the production and activation of granulocytes and macrophages to enhance immune function. Used for Pulmonary alveolar proteinosis (PAP).
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Molgramostim Open-label |
|---|---|
| Also known as | Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF) |
| Sponsor | Savara Inc. |
| Drug class | Cytokine; GM-CSF receptor agonist |
| Target | GM-CSF receptor (CD116/CD131) |
| Modality | Small molecule |
| Therapeutic area | Immunology; Rare/Orphan Diseases |
| Phase | Phase 3 |
Mechanism of action
GM-CSF is a cytokine that promotes the differentiation and proliferation of hematopoietic progenitor cells into granulocytes and macrophages, thereby increasing their numbers and enhancing their antimicrobial and antitumor activity. By boosting innate immune cell populations and function, molgramostim aims to improve immune responses in conditions characterized by immune deficiency or dysfunction.
Approved indications
- Pulmonary alveolar proteinosis (PAP)
Common side effects
- Bone pain
- Fever
- Fatigue
- Injection site reactions
- Headache
Key clinical trials
- Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP). (PHASE3)
- A Prospective, Multicenter, Open-label, Single-arm Phase II Clinical Study Evaluating the Efficacy and Safety of Spatially Fractionated Radiotherapy Combined With the PraG Strategy for the Treatment of Soft Tissue Sarcoma (PHASE2)
- Study of the Bria-IMT Regimen and CPI vs Physicians' Choice in Advanced Metastatic Breast Cancer. (PHASE3)
- Biomarker Validation Following Sargramostim Treatment in Parkinson's Disease (PHASE1)
- Sipuleucel-T Combined With Bipolar Androgen Therapy in Men With mCRPC (PHASE2)
- Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2 (PHASE3)
- Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) (PHASE3)
- Open- Label Trial of Sipuleucel-T Administered to Active Surveillance Patients for Newly Diagnosed Prostate Cancer (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Molgramostim Open-label CI brief — competitive landscape report
- Molgramostim Open-label updates RSS · CI watch RSS
- Savara Inc. portfolio CI
Frequently asked questions about Molgramostim Open-label
What is Molgramostim Open-label?
How does Molgramostim Open-label work?
What is Molgramostim Open-label used for?
Who makes Molgramostim Open-label?
Is Molgramostim Open-label also known as anything else?
What drug class is Molgramostim Open-label in?
What development phase is Molgramostim Open-label in?
What are the side effects of Molgramostim Open-label?
What does Molgramostim Open-label target?
Related
- Drug class: All Cytokine; GM-CSF receptor agonist drugs
- Target: All drugs targeting GM-CSF receptor (CD116/CD131)
- Manufacturer: Savara Inc. — full pipeline
- Therapeutic area: All drugs in Immunology; Rare/Orphan Diseases
- Indication: Drugs for Pulmonary alveolar proteinosis (PAP)
- Also known as: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing