FDA Grants Orphan Drug Status for MNV-201 in Low-Risk MDS

The FDA has granted orphan drug designation to the investigational mitochondrial cell therapy MNV-201 for the treatment of myelodysplastic syndrome (MDS).¹

This designation reinforces the novel therapeutic pathway being explored in MDS by targeting mitochondrial dysfunction, a mechanism hypothesized to underlie this age-related hematopoietic disorder. The designation for MNV-201 in MDS follows the earlier receipt of FDA fast track designation for the same indication,² highlighting the pressing unmet clinical need in this patient population.

“We continue to receive validation from the FDA for the potential of our lead product, MNV-201, this time in the form of orphan drug designation in MDS. MNV-201 targets the mitochondria, a critical multi-functional organelle. FDA designations such as [orphan drug designation] underscore the urgency of drugs treating these diseases affecting smaller populations, while providing additional benefits across the FDA process that, we expect, will prove both medically and financially valuable,” said Minovia co-founder and CEO Natalie Yivgi-Ohana, PhD, in a press release.¹

Orphan drug designation is granted to agents that fill serious unmet needs in diseases affecting less than 200,000 patients in the US. For Minovia Therapeutics, the designation for MNV-201 confers several potential advantages, including tax credits for clinical trial costs, waiver of the Prescription Drug User Fee Act (PDUFA) fee, and 7 years of market exclusivity upon marketing approval, assuming the agent maintains orphan drug status throughout the development and review process.

“Orphan drug designation for MNV-201 marks an important milestone in our mission to address critical challenges in mitochondrial health in both primary and acquired mitochondrial diseases. By leveraging our expertise in mitochondrial and hematopoietic science, and through the innovative mechanism of action of our drug product, we hope to bring forward a treatment option that could significantly improve outcomes for MDS patients,” added Minovia chief scientific officer Noa Sher, PhD, in the press release.

What is the Current Clinical Development of MNV-201?

MNV-201 is a first-in-class cell therapy leveraging Minovia's proprietary mitochondrial augmentation technology (MAT). The therapeutic mechanism involves adding healthy, energy-producing, allogeneic placental-derived mitochondria into a patient's own autologous CD34-positive hematopoietic stem and progenitor cells (HSPCs). The objective of this cellular augmentation is to restore organ function, enhance hematopoietic stability, and potentially improve overall health outcomes by addressing the root cause of mitochondrial decline.

The investigational agent is currently being evaluated in a phase 1b, open-label, dose-exploration clinical study (NCT06465160) in patients with low-risk MDS.³ The trial focuses on patients with a R-IPSS score of ≤3. The primary end points for the study include safety, as well as measures of therapeutic effect, such as an increase in hemoglobin concentration of at least 1 g/dL from baseline, a reduction in the frequency of red blood cell transfusions, and an assessment of health-related quality of life.

To be eligible for enrollment in the study, patients must have used all existing and available treatments for low-risk MDS and not be eligible for allogeneic bone marrow transplantation. Those with active infections, who are unable to undergo apheresis, a history of treatment of malignant disease (other than excision of nonmelanoma skin cancer) in the past 2 years, or who are pregnant or breastfeeding are not eligible for enrollment.

REFERENCES:

1. Minovia Therapeutics Receives FDA Orphan Drug Designation for MNV-201 in Myelodysplastic Syndrome. News release. Minovia Therapeutics. October 15, 2025. Accessed October 16, 2025. https://tinyurl.com/44jjx676

2. Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome. News release. Minovia Therapeutics. September 18, 2025. Accessed October 16, 2025. https://tinyurl.com/3stb4dpz

3. A Study to Evaluate the MNV-201 in Patients With Low Risk MDS. ClinicalTrials.gov. Updated October 1, 2025. Accessed October 16, 2025. https://clinicaltrials.gov/study/NCT06465160