MONTREAL, April 30, 2020 — ExCellThera Inc., a clinical-stage molecular medicine company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today that the European Medicines Agency has granted orphan medicinal product designation as treatment in haematopoietic stem cell transplantation to ExCellThera’s lead technology, ECT-001. ECT-001 Cell Therapy is currently the subject of several clinical trials, including two Phase II clinical trials in Canada and the United States.
Orphan medicinal product designation is granted by the European Medicines Agency (EMA) to support companies developing treatments for rare medical conditions. This designation comes with certain incentives, including a 10-year period of market exclusivity after product approval, reduced regulatory fees and protocol assistance.
“We are extremely pleased that our lead technology, ECT-001 Cell Therapy, has been granted orphan medicinal product designation from the EMA as treatment in haematopoietic stem cell transplantation,” said Dr. Guy Sauvageau, CEO and founder of ExCellThera. “The clinical data generated to date emphasizes the potential of this cell therapy to change the transplantation treatment paradigm for patients, resulting in better recovery and overall health.”
In addition to the orphan medicinal product designation granted by the European Medicines Agency (EMA), ECT-001 Cell Therapy has been granted Orphan Drug Designation for the prevention of graft-versus-host disease and Regenerative Medicine Advanced Therapy (RMAT) designation in the treatment of haematologic malignancies from the U.S. Food and Drug Administration (FDA).
ExCellThera expects that enrolment in Phase II trials of its ECT-001 Cell Therapy will be completed in the following months and intends to continue dialogue with the FDA under the auspices of the RMAT designation on a pivotal registration-enabling study, and to initiate discussions with the European Medicines Agency for the development of its technology in Europe.
About ExCellThera Inc.
ExCellThera is a clinical-stage molecular medicine 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 technology 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.