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# Orphan Status for Sickle Cell Disease Drug
- URL: https://www.fdaweb.com/orphan-status-for-sickle-cell-disease-drug/
- Published: 2024-10-25T12:00:00.000Z
- Updated: 2026-09-14T14:45:07.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158061

FDA has granted Kind Pharmaceutical an orphan drug designation for AND017 and its use in treating sickle cell disease (SCD). The company says the preclinical work supporting AND017's designation will be presented in a future scientific meeting and published in a scientific journal.

Hydroxyurea and L-glutamine are the limited FDA-approved oral treatments for SCD, and Kind says it believes AND017 might not only provide a novel oral treatment with unique mechanism of action, but also a superior safety and efficacy profile.

AND017 is described as a first-in-class hemoglobin elevating agent that targets multiple stages of the red blood cell life cycle. It is also being developed to treat anemia associated with dialysis-dependent chronic kidney disease, non-dialysis dependent chronic kidney disease, and cancer-related anemia.