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

October 16, 2025

MNV-201 has received orphan drug designation from the FDA for use in myelodysplastic syndrome.

The FDA has awarded orphan drug designation (ODD) to MNV-201 for use as a potential therapeutic option in patients with myelodysplastic syndrome (MDS), according to an announcement from Minovia Therapeutics, Ltd.1

The cell therapy leverages the company’s mitochondrial augmentation technology to add mitochondria to stem cells as a way to restore organ function and boost health. Early-phase studies have shown that this technology has provided multisystem benefits with an acceptable safety profile in Pearson syndrome. The safety and therapeutic effects of MNV-201 is under exploration in patients with low-risk MDS as part of a phase 1 study (NCT06465160) that is currently recruiting.2

“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,” Noa Sher, PhD, chief scientific officer of Minovia Therapeutics, stated in a news release.1 “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] MDS.”

MNV-201 in MDS and Beyond: Top Takeaways

The FDA granted orphan drug designation to MNV-201 for myelodysplastic syndrome (MDS), highlighting its potential to address an unmet need in this rare hematologic condition.
MNV-201 uses mitochondrial augmentation technology to enrich autologous CD34-positive cells with healthy donor mitochondria, aiming to restore cellular energy and organ function.
A phase 1b trial is enrolling patients with low-risk, transfusion-dependent MDS to assess safety, anemia improvement, and transfusion needs after MNV-201 infusion.
MNV-201 previously earned fast track designation in MDS and fast track and rare pediatric disease designations in Pearson syndrome, underscoring continued regulatory confidence in its therapeutic potential.

What Is the Design of the Early-Phase Study Evaluating MNV-201 in MDS?

The open-label, phase 1b trial is enrolling patients with low-risk MDS who have a Revised International Prognostic Scoring System score of up to 3 and mutational burden and/or low burden of high-risk mutations per Molecular International Prognostic Scoring System criteria.2 They are at least 18 years of age, have anemia and are blood transfusion dependent, and have leveraged all existing approved treatment for this disease. They could not be candidates for allogeneic bone marrow transplantation.

If they had a history of human immunodeficiency virus (HIV)-1, -2, or human T-lymphotropic virus (HTLV) I/II; active infection with hepatitis B virus, hepatitis C virus, HTLV I/II, treponema pallidum, or HIV-I-II; are not able to undergo apheresis; have a known hypersensitivity to murine proteins or iron dextran’ chronic severe infections; a disease that may put the patient at risk or interfere with study result interpretation; or a history of treatment for malignant disease in the past 2 years; they were excluded.

Patients will receive a single or repeated dose of MNV-201 for 5 days of mobilization via granulocyte colony-stimulating factor (G-CSF) once daily for 5 days. On the fifth day, after receiving the last dose of G-CSF, the patient will undergo apheresis for CD34-positive cell collection.

MNV-201 is composed of autologous CD34-positive cells that have been enriched with allogeneic mitochondria derived from healthy donor placenta. The participant’s CD34-positive cells are collected from peripheral blood after mobilization through apheresis. The donor mitochondria are aseptically isolated from placenta tissue, cryopreserved, and quality tested before use. Each treatment unit consists of a sterile, ready-to-infuse bag comprising clinical-grade MNV-201, which is prepped for intravenous administration to a single designated participant.

The primary outcome measure of the trial is the occurrence of treatment-related toxicities, and secondary outcome measures include anemia assessment, blood transfusion assessment, and quality-of-life assessment with regard to anemia.

What Should Be Taken Away With Regard to MNV-201?

“We continue to receive validation from the FDA for the potential of our lead product, MNV-201, this time in the form of ODD in MDS. MNV-201 targets the mitochondria, a critical multi-functional organelle,” co-founder and chief executive officer, Natalie Yivgi-Ohana, PhD, of Minovia, stated in the news release.1 “FDA designations such as ODD 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.”

MNV-201 previously received a fast track designation from the FDA for MDS, as well as fast track and rare pediatric disease designations in Pearson syndrome.

References

Minovia Therapeutics receives FDA orphan drug designation for MNV-201 in myelodysplastic syndrome. News release. Minovia Therapeutics, Ltd. October 15, 2025. Accessed October 15, 2025. https://www.globenewswire.com/news-release/2025/10/15/3166966/0/en/Minovia-Therapeutics-Receives-FDA-Orphan-Drug-Designation-for-MNV-201-in-Myelodysplastic-Syndrome.html
A study to evaluate the MNV-201 in patients with low-risk MDS. ClinicalTrials.gov. Updated October 1, 2025. Accessed October 15, 2025. https://clinicaltrials.gov/study/NCT06465160