MNV-201 receives fast track, rare pediatric disease to treat Pearson Syndrome

MNV-201 receives fast track, rare pediatric disease to treat Pearson Syndrome | Image Credit: © John Doe - © John Doe - stock.adobe.com.

The FDA has granted both fast track designation and rare pediatric disease designations to Minovia Therapeutics Ltd's lead investigational compound MNV-201, which is being evaluated in phase 2 clinical trials to treat Pearson Syndrome. According to a press release from the company, the rare and life-threatening disorder is caused by large-scale deletions in mitochondrial DNA that impair the energy-generating function of cells, which leads to bone marrow failure, metabolic crises, and organ dysfunction.¹

MNV-201 is a first-in-class cell therapy that, according to the company, adds "healthy, energy-producing mitochondria into a patient’s own stem cells — aiming to restore organ function and improve health." Minovia stated that its proprietary Mitochondrial Augmentation Technology (MAT) has demonstrated a strong safety profile in early-stage clinical studies, as well as provided signs of multi-system benefit in patients with Pearson Syndrome. These benefits have included improvements in growth, muscle function, hematologic stability, and improved quality of life.

“Both Fast Track Designation and Pediatric Rare Disease Designation are critical milestones for Minovia, as they strongly validate the clinical approach for our science, while also acknowledging the urgent need for new treatment options for Pearson Syndrome," said Natalie Yivgi-Ohana, PhD, Minovia's co-founder and CEO. "Importantly, these FDA designations help us to decrease the potential time to market and provide additional benefits across the FDA process that will prove both medically and financially valuable."

Currently, there are no cures for Pearson Syndrome, which impacts the bone marrow, pancreas, and other organs such as the liver. The condition is typically diagnosed in infancy, according to The Cleveland Clinic. The condition can also cause issues with the blood, such as shortages of red blood cells, white blood cells, and platelets.²

Symptoms can include fatigue and weakness, easy bruising, frequent bouts of diarrhea, infections, pale skin, vomiting, and growth faltering, among others. According to Cleveland Clinic, a majority of Pearson Syndrome cases occur for unknown reasons, though it can be inherited. Still, these cases are rare and not well understood.

References:

1. Minovia Therapeutics announces FDA fast track and rare pediatric disease designations for MNV-201 in Pearson Syndrome. Minovia Therapeutics. Press release. June 30, 2025. Accessed June 30, 2025.
2. Pearson Syndrome. Cleveland Clinic. Updated April 14, 2024. Accessed June 30, 2025. https://my.clevelandclinic.org/health/diseases/pearson-syndrome