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# Orphan Status for Minovia Myelodysplastic Drug
- URL: https://www.fdaweb.com/orphan-status-for-minovia-myelodysplastic-drug/
- Published: 2025-10-16T12:00:00.000Z
- Updated: 2026-09-14T15:24:16.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160058

FDA has granted Minovia Therapeutics an orphan drug designation for its lead investigational therapy, MNV-201, for myelodysplastic syndrome (MDS). MNV-201 is described by the company as a first-in-class mitochondrial cell therapy that uses Minovia’s Mitochondrial Augmentation Technology to introduce healthy mitochondria into a patient’s stem cells, aiming to restore cellular function. Early-stage studies in Pearson Syndrome patients have shown a favorable safety profile and signs of multi-system benefit, including improved hematologic stability and quality of life, the company says.

MDS is a serious age-related blood disorder characterized by ineffective hematopoiesis and a risk of progression to acute myeloid leukemia, according to Minovia. The median age at diagnosis is about 70, and the disease carries a high symptom burden and risk of mortality, it says. The company is currently conducting a Phase 1b study in low-risk MDS patients, with six of nine planned patients already dosed.