Hope Biosciences Earns FDA RMAT Status for Stem Cell Therapy in Relapsing-Remitting MS

Hope Biosciences, a U.S.-based stem cell banking and therapeutic manufacturer, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for its adipose-derived mesenchymal stem cells (HB-adMSCs) to treat relapsing-remitting multiple sclerosis (RRMS).

The RMAT designation, a significant regulatory milestone, was granted based on promising results from a Phase II clinical trial conducted by Hope Biosciences Research Foundation (HBRF), a nonprofit based in Sugar Land, Texas. RMAT status is reserved for therapies targeting serious or life-threatening conditions and showing preliminary clinical evidence of substantial improvement over existing treatments. The designation allows for expedited development and enhanced FDA collaboration, including priority review and eligibility for accelerated approval.

“Receiving the first RMAT designation for multiple sclerosis is a tremendous acknowledgment of our technology’s potential to impact the world of debilitating autoimmune conditions,” said Donna Chang, CEO of Hope Biosciences. She credited the HBRF team for their clinical expertise and patient-centered approach.

The pivotal Phase II trial (NCT05116540), conducted from 2023 to 2024, was the first in the world to deliver high-dose pure mesenchymal stem cells to MS patients over repeated treatments. The double-blind, randomized study involved 24 participants, with the treatment group receiving six infusions of 200 million stem cells each—totaling 1.2 billion cells over 32 weeks.

According to Dr. Ridhima Vij, Clinical Research Scientist at HBRF, the study met its primary endpoint, showing statistically significant improvements in both physical and mental health among patients receiving HB-adMSCs. These gains were measured using the MS Quality of Life-54 Instrument, which evaluates symptoms such as fatigue, mobility, cognition, and pain—factors that deeply affect daily life for MS patients.

MS is a chronic autoimmune disorder affecting over 2 million people globally, including around 400,000 in the U.S. Relapsing-remitting MS, the most common form, involves episodes of symptom flare-ups followed by periods of remission. The disease often begins in early adulthood and disproportionately affects women. It also poses a heavy economic burden, with estimated lifetime treatment costs nearing $5 million per patient.

“We are grateful to the FDA for recognizing the urgent need for new MS treatments,” said Chang. “We remain focused on getting this therapy to patients quickly and look forward to working with the agency on the next steps, including filing a Biologics License Application.”

Hope Biosciences also confirmed its invitation to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot program, which aims to accelerate regulatory review by ensuring early alignment on manufacturing requirements.