Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it may allow them to apply for faster review and approval and get their therapy to more patients faster.

Here’s why that RMAT designation matters.

Kidney disease

Over 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. The best available treatment for these patients is a kidney transplant from a genetically matched living donor.

However, patients who receive a transplant must take lifelong immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer, and diabetes.  Despite these drugs, many patients still lose transplanted organs due to rejection.

To tackle this problem, Medeor is developing a stem cell-based therapy called MDR-101. This is being tested in a Phase 3 clinical trial, and it’s hoped it will eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.

Stem cells

The company takes blood-forming stem cells and immune cells from the organ donor and infuses them into the patient receiving the donor’s kidney. Introducing the donor’s immune cells into the patient creates a condition called “mixed chimerism,” where immune cells from the patient and the donor can co-exist. In this way, the patient’s immune system can adapt to and tolerate the donor’s kidney, potentially eliminating the need for immunosuppressive drugs normally required to prevent transplant rejection.

So how does RMAT designation help? The FDA created the RMAT program to speed up the development and review of regenerative medicine therapies that can treat, modify, reverse, or cure a serious condition. If MDR-101shows it is both safe and effective, RMAT could help it get faster approval for wider use.

In a news release, Giovanni Ferrara, President and CEO of Medeor, welcomed the news.

“This important designation underscores the tremendous unmet medical need for alternatives to today’s immunosuppressive therapies for transplantation. We have the potential to help people live longer, healthier lives without the need for high-dose and chronic immunosuppression, and we thank the FDA for this designation that will assist us in progressing as efficiently as possible toward a commercially available product.”

This is the seventh CIRM-supported project to receive RMAT designation. The others are jCyte, Lineage, Humacyte, St. Jude’s/UCSF X-linked SCID, Poseida, Capricor