MGTA-456 Earns FDA’s Regenerative Medicine Advanced Therapy Designation

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The FDA created (RMAT) designation to advance the development and approval of potential therapies. They designate products that demonstrate clinical data to treat, modify, or cure a serious or fatal disease, and focus on unmet medical needs.

“This RMAT designation was based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative, life-saving potential of MGTA-456 for children suffering from inherited metabolic disorders,” John Davis, MD, chief medical officer at Magenta Therapeutics, said in a press release.

In fact, MGTA-456 is a stem cell treatment, allogenic hematopoietic stem cell therapy (HSCT)  in particular is created to yield the growth of inherited metabolic disorders.

Practitioners deliver a high-dose of stem cells to reboost the patient’s immune system. They conduct a technique where a genetically identical donor (allogenic), donates stem cells that are transplanted into the patient.

Furthermore, Magenta is developing MGTA-456 as a treatment for multiple diseases. Magenta is doing a Phase 2 clinical trial (NCT03406962) in patients with various genetic metabolic disorders.

Moreover, this trial is being conducted at Cincinnati Children’s Hospital, Duke University, Emory University and the University of Minnesota. Patients improved from a previous trial where other therapies did not help. Patients with adrenoleukodystrophy (cALD), showed reduced brain inflammation on MRI scans.

“We look forward to collaborating closely with the FDA as we seek to rapidly advance MGTA-456 through the ongoing Phase 2 study, and into potential pivotal studies in 2020,” Davis said.

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