ECT-001 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation By U.S. FDA

The potential new multiple myeloma therapy ECT-001 has been designated a Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA).
The RMAT designation is very similar in significance to the better known Breakthrough Therapy designation, which also is granted by the FDA. Both the RMAT and Breakthrough Therapy designations are intended to shorten the time it takes important new therapies to gain FDA marketing approval.
The key difference between the two designations is that the RMAT designation is for cell-based therapies, while the Breakthrough Therapy designation is for drug-based therapies.
ECT-001 consists of what are known as “expanded cord blood stem cells.” Patients who are treated with ECT-001 undergo the equivalent of a standard allogeneic (donor) stem cell transplant, except that expanded cord blood stem cells are used during the transplant instead of stem cells from adult donors.
Cord blood stem cells are stem cells harvested from the blood present in umbilical cords and placentas. Such blood is often collected after babies are born, after which it is stored in cord blood banks.
Expanded cord blood stem cells are cord blood stem cells that have been cultured and encouraged to replicate (“expand”) in significant numbers.
An important upside of cord blood stem cells is that using them during allogeneic stem cell transplants has been shown to reduce the risk and severity of graft-versus-host disease (GVHD), a serious, sometimes fatal, complication of allogeneic transplants.
The downside to cord blood stem cells is that the number of them available to be administered to a patient during a transplant is much lower than is the case with stem cells from non-cord blood sources. As a result, patients receiving cord blood stem cell transplants must wait longer after transplantation for their blood counts to return to normal levels, leaving them at greater risk for infection
By using expanded cord blood stem cells, researchers can transplant patients with a greater number of stem cells, with the goal of reducing post-transplant infection risk while still providing patients the benefits associated with cord blood stem cell transplantation.
The RMAT designation for ECT-001 is for the treatment of blood cancers, including multiple myeloma. Results of an initial clinical trial of ECT-001 in blood cancer patients were presented at the 2018 American Society of Hematology (ASH) annual meeting (abstract), and a trial testing ECT-001 in high-risk newly diagnosed multiple myeloma patients is ongoing in Montreal, Quebec.
ExCellThera Inc., the Canadian company that is developing ECT-001, announced the ECT-001 RMAT designation in a press release issued last week.
ECT-001 is only the second potential new myeloma therapy currently known to have been granted an RMAT designation. Last November, Poseida Therapeutics announced that P-BCMA-101, a chimeric antigen receptor (CAR) T-cell therapy it is developing as a potential treatment for multiple myeloma, had been granted an RMAT designation by the FDA (related press release).
(The FDA does not publicly announce RMAT designations, and companies are not legally required to report the designation beyond any obligation they may have as a result of financial reporting regulations.)
More About ECT-001 And Cord Blood Stem Cells
ECT-001 cells are produced by placing cord blood stem cells for seven days in a medium that contains UM171, a proprietary compound developed by ExCellThera explicitly for the purpose of encouraging the replication (expansion) of cord blood stem cells.
In the clinical trial of ECT-001 whose results were reported at last year’s ASH meeting, exposing cord blood stem cells to UM171 over the course of a week led to a median 35-fold increase in the number of stem cells available to be administered to each trial participant.
This increased stem cell dose is the goal of the expansion process, as it should help reduce the period of time after transplantation that patients receiving the stem cells have very low white blood cell counts. This, in turn, should reduce the risk of serious infection during that time.
More About RMAT Designations
RMAT and Breakthrough Therapy designations both make it possible for companies to have earlier, more frequent, and more senior-level interactions with the FDA about the qualifying therapies. The designations also allow companies to submit approval applications for the therapies on a rolling basis, rather than having to wait until all information for an application is available before it can be submitted.
For a cell-based therapy to qualify for an RMAT designation, it must be intended to treat, modify, reverse, or cure a serious disease or condition, and there must be preliminary clinical evidence that the therapy addresses an unmet medical need related to the disease or condition.
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