ECT-001 Granted Regenerative Medicine Advanced Therapy Designation for Treatment of Hematologic Malignancies
Posted: Friday, May 31, 2019
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECT-001 in the treatment of hematologic malignancies. The RMAT designation is based on data from phase I/II clinical trials using ECT-001 (ClinicalTrials.gov identifier NCT03441958) to expand stem and immune cells for the treatment of blood cancers.
The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology, reportedly capable of expanding the number of stem and immune cells in up to 7 days, is used in cord blood transplant therapies for patients with blood cancers. ECT-001 has been reported to reduce the incidence of transplant-related mortality, the risk of chronic graft-versus-host disease, and the risk of relapse.
Various clinical studies using ECT-001 are currently ongoing in the treatment of multiple myeloma, high-risk leukemia, and other hematologic malignancies. ECT-001 has also received FDA Orphan Drug designation for the prevention of graft-versus-host disease.
