MNV-201 Secures FDA Fast Track Designation in Myelodysplastic Syndrome

The U.S. Food and Drug Administration (FDA) has granted fast track designation to the investigational therapy MNV-201 for the treatment of myelodysplastic syndrome (MDS), according to a news release from Minovia Therapeutics Ltd., a clinical-stage biotechnology company.

This designation highlights the potential of the first-in-class mitochondrial cell therapy for patients with this serious age-related blood disorder. The company previously received FDA fast track and rare pediatric disease designations for MNV-201 in Pearson Syndrome, an ultra-rare mitochondrial disorder affecting children, which is currently being studied in a phase 2 clinical trial.

Dr. Natalie Yivgi-Ohana, co-founder and CEO of Minovia, stated, “The fast track designation in MDS validates the urgency and potential of our mitochondrial-targeting therapy. FDA designations like this can help accelerate development and provide additional regulatory benefits, which we expect will be valuable for both patients and the company.”

Fast track status allows for increased FDA engagement, eligibility for priority review and the option for rolling submission of a biologics license application for MNV-201, the news release explained.

“Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast track addresses a broad range of serious conditions. …Determining whether a condition is serious is generally is based on whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one,” the U.S. FDA explained on their official website.

Understanding Myelodysplastic Syndrome and Patient Impact

MDS is a group of disorders characterized by ineffective hematopoiesis, which leads to low blood cell counts (cytopenia) and a risk of progression to acute myeloid leukemia (AML). Patients often experience fatigue, infection risk, anemia and bleeding, all of which significantly impact quality of life. The median age at diagnosis is approximately 70 years, although MDS can appear in younger patients with Pearson Syndrome, a condition in which about 15% develop sideroblastic anemia, a common symptom associated with mitochondrial dysfunction.

The primary goals of treatment for MDS are to improve blood counts, reduce symptoms, lower the risk of progression to AML and enhance both lifespan and quality of life. Minovia has developed novel blood biomarkers to assess mitochondrial health and has provided evidence that MDS may be, in part, an age-related mitochondrial disorder. These insights have informed the ongoing phase 1b study of MNV-201 in patients with low-risk MDS, with six of nine planned patients already dosed.

How MNV-201 Works and What It Means for Patients

MNV-201 is a mitochondrial cell therapy that employs Minovia’s proprietary Mitochondrial Augmentation Technology. The treatment introduces healthy, energy-producing mitochondria into a patient’s own stem cells, aiming to restore organ function and improve overall health. Early clinical studies in Pearson Syndrome patients have demonstrated a strong safety profile and signs of multi-system benefits, including improvements in growth, muscle function, blood stability and quality of life.

For patients with MDS, MNV-201 represents a novel approach that targets a core cellular mechanism underlying the disease rather than only treating symptoms. By addressing mitochondrial dysfunction, this therapy could help stabilize blood counts, reduce complications, and potentially slow or prevent progression to AML. The FDA fast track designation may help bring this therapy to patients faster while allowing for closer collaboration between Minovia and regulators to address the urgent medical need.

With MNV-201 advancing through clinical trials and the FDA recognizing its potential through fast track status, patients with MDS and related mitochondrial disorders may have new treatment options on the horizon, the news release concluded. The therapy underscores the potential of mitochondrial-targeted approaches in improving both outcomes and quality of life for patients facing serious, age-related blood diseases.

References

1. “Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome,” by Minovia Therapeutics. News release; Sept. 18, 2025.
2. “Fast Track,” by the U.S. Food and Drug Administration. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

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