FDA Fast Tracks MNV-201 in Myelodysplastic Syndromes

The FDA has granted fast track designation to MNV-201 for the treatment of myelodysplastic syndromes (MDS).¹

FDA fast track is intended to expedite the development of therapies that fill serious unmet medical needs. With this designation, Minovia Therapeutics, the sponsor, has the opportunity for increased FDA interactions and eligibility for priority review and rolling submission of a biologics license application.

“The fast track designation in MDS is further validation of the urgency and potential for our science, which targets the mitochondria, a critical multi-functional organelle. FDA designations such as the [fast track designation] help us to decrease the potential time to market and provide 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.

MNV-201 also received fast track and rare pediatric disease designation for the treatment of Pearson Syndrome, an ultrarare mitochondrial disorder affecting pediatric patients.

MNV-201 is being investigated in a phase 1 study (NCT06465160) in patients with low-risk MDS.² The study’s primary end point is incidence of treatment-related adverse events, and secondary end points include anemia assessment, blood transfusion assessment, and health-related quality of life.

Patients are required to have low-risk MDS with an R-IPSS score of at least 3 with mutational burden or a low burden of high-risk mutations defined by IPSS-M. Patients are also required to have anemia and be blood transfusion-dependent and utilized all available treatments. Further, patients cannot be eligible for allogeneic bone marrow transplantation, a history of treatment with gene therapy, or current active infection.

The study treatment consists of a single or repeated dose of MNV-201 by infusion after 5 days of mobilization by granulocyte colony stimulating factor (G-CSF) and apheresis to collect CD34-positive cells. MNV-201 is comprised of the autologous CD34-positive cells enriched with allogeneic placenta-derived mitochondria.

The study has an estimated enrollment of 15 patients and a planned completion date of December 31, 2029.

REFERENCES:

1. Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome. News release. Minovia Therapeutics. September 18, 2025. Accessed September 19, 2025. https://tinyurl.com/3stb4dpz

2. A Study to Evaluate the MNV-201 in Patients with Low Risk MDS. ClinicalTrials.gov. Updated March 17, 2025. Accessed September 19, 2025.https://clinicaltrials.gov/study/NCT06465160