FDA Grants Orphan Drug Designation to MNV-201 for Myelodysplastic Syndrome

The U.S. Food and Drug Administration has granted orphan drug designation to the investigational therapy MNV-201 for patients with myelodysplastic syndrome, a serious, age-related blood disorder that can progress to acute myeloid leukemia (AML), according to a news release from Minovia Therapeutics.

Orphan drug designation, according to the release, is granted to drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. The designation not only acknowledges the unmet need for new myelodysplastic syndrome treatments but also offers regulatory and financial benefits to encourage the development of promising therapies. These may include tax credits, market exclusivity, and guidance from the FDA throughout the clinical process.

This new designation adds to MNV-201’s existing fast track designation in Myelodysplastic Syndrome. Notably, the U.S. FDA website states that fast track designation is intended to speed up the development and review of drugs for serious conditions with unmet medical needs, helping patients access important new treatments sooner.

“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 myelodysplastic syndrome. 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.

FDA Designation Highlights the Use of MNV-201 for Rare Blood Cancer

MNV-201 is a first-in-class cell therapy built on Minovia’s Mitochondrial Augmentation Technology (MAT) platform. The therapy involves enhancing a patient’s own stem cells with healthy, energy-producing mitochondria. By restoring mitochondrial function, the agent aims to improve organ function, stabilize blood cell production, and enhance overall health.

“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 patients [with] myelodysplastic syndrome,” Minovia chief scientific officer, Noa Sher, added.

In earlier studies, MNV-201 demonstrated a strong safety profile and encouraging signs of multi-system benefit in patients with Pearson Syndrome, including improved growth, muscle function, hematologic stability, and quality of life.

What is Myelodysplastic Syndrome?

Myelodysplastic syndrome is a group of blood disorders caused by ineffective production of blood cells in the bone marrow. The condition is associated with cytopenia, or low blood counts, which can lead to fatigue, infection, or bleeding complications. It also carries a risk of progression to acute myeloid leukemia, a more aggressive form of blood cancer. The goals of treatment include improving blood counts, managing symptoms, and reducing disease progression.

The median age at diagnosis for myelodysplastic syndrome is approximately 70 years, though some patients with Pearson Syndrome, which is a rare inherited mitochondrial disorder, develop myelodysplastic syndrome at higher rates. Notably, about 15% of patients with myelodysplastic syndrome present with sideroblastic anemia, a symptom often seen in Pearson Syndrome.

Minovia’s research team has developed novel blood biomarkers to measure mitochondrial health and was the first to demonstrate evidence suggesting that myelodysplastic syndrome may be an age-related mitochondrial disease. The company is currently conducting a phase 1b clinical study of MNV-201 in low-risk patients with myelodysplastic syndrome, with six of nine planned participants already enrolled and dosed.

References

1. “Minovia Therapeutics Receives FDA Orphan Drug Designation for MNV-201 in Myelodysplastic Syndrome,” by Minovia Therapeutics Ltd. News release; Oct. 16, 2025.
2. “Fast Track,” by U.S. FDA. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

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