Stem Cell Therapeutics Corp. has announced that it has been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the use of tigecycline in the treatment of acute myeloid leukemia (AML).
Orphan Drug designation is granted to therapeutics treating rare diseases affecting less than 200,000 people in the U.S. The designation entitles the sponsor to seven years of market exclusivity as well as opportunities for additional funding and expert protocol assistance.
“Orphan Drug status significantly enhances the commercial potential of tigecycline in AML, a disease which is notoriously difficult to treat", said the company’s Vice President, Drug Development, Dr. Penka Petrova.
Petrova continued, “Through its unique mechanism of action and synergy with existing AML therapies, tigecycline has the potential to positively impact the standard of care in this disease”.
The company’s program is based on Dr. Aaron Schimmer’s published findings that tigecycline, an FDA-approved antibiotic, selectively targets leukemia cells and leukemic stem cells by inhibiting mitochondrial protein synthesis and thus shutting down the cells’ energy supply.
A Phase I Canadian and U.S. multicenter dose-escalation clinical trial in patients with relapsed or refractory AML is nearing completion.
“The Orphan Drug designation covers tigecycline as an active ingredient and is independent of the drug formulation,” added Dr. Bob Uger, the company’s Chief Scientific Officer. “We are investigating improved formulations of tigecycline, which we believe will be critical for the commercialization of this technology”.