SCT’s AML Treatment Gets Orphan Status

Stem Cell Therapeutics Corp. has received Orphan Drug designation from the FDA for the use of tigecycline in the treatment of acute myeloid leukemia (AML).

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.

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.