Canadian biopharmaceutical development firm Stem Cell Therapeutics (SCT) has received an orphan drug designation from the US Food and Drug Administration (FDA) for the use of tigecycline to treat acute myeloid leukaemia (AML) patients.
SCT vice-president of drug development Penka Petrova said: "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."
According to the company, the designation is expected to improve the commercial potential of tigecycline in AML, which is a particularly difficult to treat disease.
The designation now entitles the company to seven years of market exclusivity, as well as opportunities for additional funding and expert protocol assistance.
The company’s programme is based on Dr Aaron Schimmer’s published results that tigecycline, selectively targets leukaemia cells and leukaemic stem cells by inhibiting mitochondrial protein synthesis and shuts down the cells’ energy supply.
The company said that a Phase I Canadian and US multicentre dose-escalation clinical trial in patients with relapsed or refractory AML is nearing completion.
Stem Cell Therapeutics chief scientific officer Bob Uger said the orphan drug status covers tigecycline as an active ingredient and is independent of the drug formulation.
"We are investigating improved formulations of tigecycline, which we believe will be critical for the commercialisation of this technology," Uger said.
The cancer stem cell (CSC) concept advocates that the growth of tumours is driven by a rare population of dedicated cells that have stem cell-like properties, including self-renewal.
SCT is an immuno-oncology firm advancing cancer stem cell discoveries into new cancer therapies and it has two premier preclinical programmes, SIRPaFc and a CD200 monoclonal antibody (mAb), which target two key immunoregulatory pathways that tumour cells exploit to evade the host immune system.
Image: Myeloblasts with Auer rods seen in Acute Myeloid Leukemia (AML). Photo: courtesy of Paulo Mourao.
