Biomarker and diagnostic research for autoimmune pulmonary alveolar proteinosis has been reported in the published literature.

No approved treatments are currently available for autoimmune pulmonary alveolar proteinosis. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for autoimmune pulmonary alveolar proteinosis, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for autoimmune pulmonary alveolar proteinosis. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Gene therapy approaches for autoimmune pulmonary alveolar proteinosis have been reported in the published literature.

Phenotype severity distribution: 3 always present features.

Estimated prevalence: 1-9 in 1,000,000 (Rare).

4 clinical trials registered, 1 recruiting. Interventions under study include drug therapy. Pipeline includes 2 PHASE3, 2 PHASE2. Research is sponsored by a mix of industry and academic institutions.

150 publications have been identified in PubMed for autoimmune pulmonary alveolar proteinosis. Research spans Case Report / Case Series (42%), Review / Meta-Analysis (20%), and Epidemiology / Natural History (12%).

Recent Publications

• Lian L (2026). [PMID: 41671176](https://pubmed.ncbi.nlm.nih.gov/41671176/). *Proceedings of the National Academy of Sciences of the United States of America*. [Case Report / Case Series]

• Song X (2026). [PMID: 42119776](https://pubmed.ncbi.nlm.nih.gov/42119776/). *Chest*. [Epidemiology / Natural History]

• Fujishiro E (2026). [PMID: 42250564](https://pubmed.ncbi.nlm.nih.gov/42250564/). *Respir Investig*. [Epidemiology / Natural History]

• Dang M (2026). [PMID: 42045620](https://pubmed.ncbi.nlm.nih.gov/42045620/). *Lung*. [Review / Meta-Analysis]

• Naeem U (2026). [PMID: 41742124](https://pubmed.ncbi.nlm.nih.gov/41742124/). *BMC pulmonary medicine*. [Review / Meta-Analysis]

• Fujita S (2026). [PMID: 41649888](https://pubmed.ncbi.nlm.nih.gov/41649888/). *Immunol Med*. [Case Report / Case Series]

• Sharma S (2026). [PMID: 41835822](https://pubmed.ncbi.nlm.nih.gov/41835822/). *Indian J Thorac Cardiovasc Surg*. [Case Report / Case Series]

• Carrington JM (2026). [PMID: 29493933](https://pubmed.ncbi.nlm.nih.gov/29493933/). *Unknown Journal*. [Review / Meta-Analysis]

• Hou SX (2026). [PMID: 42236457](https://pubmed.ncbi.nlm.nih.gov/42236457/). *Zhonghua Jie He He Hu Xi Za Zhi*. [Case Report / Case Series]

• Sugino K (2026). [PMID: 41815743](https://pubmed.ncbi.nlm.nih.gov/41815743/). *Respirol Case Rep*. [Epidemiology / Natural History]