Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for chronic lymphocytic leukemia/small lymphocytic lymphoma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Gene therapy approaches for chronic lymphocytic leukemia/small lymphocytic lymphoma have been reported in the published literature.

184 publications have been identified in PubMed for chronic lymphocytic leukemia/small lymphocytic lymphoma. Research spans Review / Meta-Analysis (24%), Case Report / Case Series (22%), and Epidemiology / Natural History (22%).

Recent Publications

• Pinilla-Ibarz J (2026). [PMID: 41662512](https://pubmed.ncbi.nlm.nih.gov/41662512/). *Expert Rev Hematol*. [Epidemiology / Natural History]

• Woyach JA (2026). [PMID: 41353787](https://pubmed.ncbi.nlm.nih.gov/41353787/). *J Clin Oncol*. [Clinical Trial Publication]

• Jurczak W (2026). [PMID: 41363773](https://pubmed.ncbi.nlm.nih.gov/41363773/). *J Clin Oncol*. [Clinical Trial Publication]

• Wierda WG (2026). [PMID: 41825137](https://pubmed.ncbi.nlm.nih.gov/41825137/). *J Natl Compr Canc Netw*. [Review / Meta-Analysis]

• Tidswell R (2026). [PMID: 41698679](https://pubmed.ncbi.nlm.nih.gov/41698679/). *Eur J Haematol*. [Epidemiology / Natural History]

• D'Amato M (2026). [PMID: 41793543](https://pubmed.ncbi.nlm.nih.gov/41793543/). *Clin Exp Med*. [Review / Meta-Analysis]

• Ghosh N (2026). [PMID: 41529988](https://pubmed.ncbi.nlm.nih.gov/41529988/). *Clin Lymphoma Myeloma Leuk*. [Review / Meta-Analysis]

• Slater VE (2026). [PMID: 42066411](https://pubmed.ncbi.nlm.nih.gov/42066411/). *Leuk Res*. [Diagnostic / Biomarker]

• Sun M (2026). [PMID: 42000210](https://pubmed.ncbi.nlm.nih.gov/42000210/). *Clin Lymphoma Myeloma Leuk*. [Clinical Trial Publication]

• Allan JN (2026). [PMID: 41627367](https://pubmed.ncbi.nlm.nih.gov/41627367/). *Adv Ther*. [Review / Meta-Analysis]