The EMA orphan drug status provides a number of benefits, including ten years of market exclusivity from product launch in the EU, fee reductions, and access to the central authorization procedure.
The company has also been granted orphan drug designation for marizomib in multiple myeloma by the US Food and Drug Administration (FDA).
Triphase founder, president and CEO Frank Stonebanks said securing orphan designation for marizomib in the EU for multiple myeloma represents an important milestone in the development of this novel, highly potent proteasome inhibitor.
"We are committed to developing this promising agent through proof-of-concept to fill an unmet medical need for patients with relapsed and refractory multiple myeloma, an often fatal hematologic cancer for which new treatment options are needed," Stonebanks said.
Marizomib is a new, highly potent proteasome inhibitor that irreversibly targets and inhibits all three proteasome subunits, allowing for more durable and sustained responses.
The company is developing marizomib in both intravenous (IV) and oral formulations for hematologic malignances and solid tumors.
The drug has showed activity in a Phase I trial in patients with multiple myeloma refractory to lenalidomide or bortezomib.