Minovia Therapeutics said that the U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to its investigative therapy MNV-201 for treatment of myelodysplastic syndrome (MDS). The designation complements existing Fast Track status for MDS and previous designations in Pearson Syndrome according to the company.
The company noted that ODD provides incentives such as tax credits and market exclusivity, aimed at encouraging development of treatments for rare diseases. Minovia also announced it will merge with Launch One Acquisition Corp., creating a Nasdaq-listed mitochondrial therapy business, with the combined entity expected to trade under a new ticker in late 2025.
MDS is a hematopoietic disorder characterized by ineffective blood cell production, cytopenias, and a risk of progression to acute myeloid leukemia. Median age at diagnosis is around 70 years. The company said that in its ongoing Phase Ib trial in low-risk MDS, six of nine expected patients have been dosed so far.
MNV-201 is a cell therapy leveraging Minovia’s proprietary Mitochondrial Augmentation Technology (MAT), which enriches autologous stem cells with healthy mitochondria. In early-stage clinical studies in Pearson Syndrome, MAT has shown safety and signs of multi-system benefit including improvements in growth, muscle function, hematologic stability, and quality of life, the company claims.
Minovia described the ODD grant as a key regulatory milestone in its mission to bring mitochondrial therapies to patients with primary and acquired mitochondrial dysfunction.
