Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for hairy cell leukemia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

vemurafenib is referenced in active clinical trials for hairy cell leukemia (designated 2014).

Gene therapy approaches for hairy cell leukemia have been reported in the published literature.

125 publications have been identified in PubMed for hairy cell leukemia. Research spans Case Report / Case Series (37%), Review / Meta-Analysis (23%), and Epidemiology / Natural History (12%).

Recent Publications

• Chen Z (2026). [PMID: 42026747](https://pubmed.ncbi.nlm.nih.gov/42026747/). *Anticancer Drugs*. [Case Report / Case Series]

• Singi S (2026). [PMID: 41981246](https://pubmed.ncbi.nlm.nih.gov/41981246/). *Commun Med (Lond)*. [Gene Therapy / Novel Therapeutics]

• Zhang G (2026). [PMID: 41296002](https://pubmed.ncbi.nlm.nih.gov/41296002/). *Blood Adv*. [Review / Meta-Analysis]

• Blombery P (2026). [PMID: 41528825](https://pubmed.ncbi.nlm.nih.gov/41528825/). *Leuk Lymphoma*. [Diagnostic / Biomarker]

• Alameddine Z (2026). [PMID: 42005217](https://pubmed.ncbi.nlm.nih.gov/42005217/). *Cureus*. [Case Report / Case Series]

• Watrin T (2026). [PMID: 41048115](https://pubmed.ncbi.nlm.nih.gov/41048115/). *Leuk Lymphoma*. [Review / Meta-Analysis]

• Veiga LD (2026). [PMID: 41497420](https://pubmed.ncbi.nlm.nih.gov/41497420/). *Cureus*. [Case Report / Case Series]

• Rehman A (2026). [PMID: 42109837](https://pubmed.ncbi.nlm.nih.gov/42109837/). *Case Rep Med*. [Case Report / Case Series]

• Chen C (2026). [PMID: 41974312](https://pubmed.ncbi.nlm.nih.gov/41974312/). *Hum Pathol*. [Epidemiology / Natural History]

• Chandna S (2026). [PMID: 41552793](https://pubmed.ncbi.nlm.nih.gov/41552793/). *Respir Med Case Rep*. [Case Report / Case Series]