ExCellThera receives FDA orphan drug designation for ECT-001 for the prevention of graft-versus-host disease
Posted on December 17th, 2018
MONTREAL, December 17, 2018 – ExCellThera Inc., a biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today that the U.S. Food and Drug Administration (FDA) has granted the ExCellThera’s lead solution, ECT-001, orphan drug designation for the prevention of graft-versus-host-disease (GvHD). ECT-001 uses a proprietary small molecule and an optimized culture system to expand therapeutic stem and immune cells and is being evaluated in several clinical trials.
Orphan drug designation is granted by the FDA’s Office of Orphan Drug Products to support companies developing treatments that are expected to provide significant therapeutic advantage over existing treatments, and that target rare medical conditions. With this designation, ExCellThera gains certain incentives, including seven-year market exclusivity, exemption from certain fees and fast-tracking of regulatory proceedings.
“Preliminary results from clinical trials using ECT-001 as a novel treatment for blood cancers have been extremely encouraging, with a significant reduction in the incidence and severity of chronic GvHD. Receiving orphan drug designation is yet another important milestone to speed advancement to the clinic,” said Dr. Guy Sauvageau, CEO and founder of ExCellThera. “We believe that ECT-001 has the potential to change the stem cell therapy paradigm for better patient recovery and health.”
GvHD is a life-threatening complication of certain cell therapies and transplants, in which the grafted cells (from a donor) attack the host, leading to a severe immune response. GvHD occurs when the transplanted cells are not a perfect match to the patient.
The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology, capable of expanding the number of stem and immune cells exponentially in as little as seven days, is used in novel curative cord blood transplant therapies for patients with blood cancers, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients.
About ExCellThera Inc.
ExCellThera is a clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellThera’s lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials.