FDA Grants Orphan Status To Triphase Cancer Drug Marizomib
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Triphase Accelerator’s cancer treatment Marizomib. The designation gives Triphase seven-year marketing exclusivity for marizomib once the FDA approves the drug.
Frank Stonebanks, Founder, President and CEO of Triphase, said, “We are pleased that the FDA has granted orphan drug designation for the development of marizomib to benefit patients with multiple myeloma. While patients with refractory multiple myeloma are living longer and better lives as a result of medical innovation, there is still a need for new treatment options. We are excited to move forward with the development of marizomib, a potential best-in-class agent, and hope to advance the treatment paradigm that will turn this once acute disease into a long-term manageable disease.”
Marizomib is a novel proteasome inhibitor currently undergoing evaluation for the treatment of multiple myeloma and other cancer indications. A marizomib intravenous formulation has been assessed in more than 230 patients across four Phase I and II studies, either as a single agent or in combination with dexamethasone or an HDAC inhibitor. The company is at present evaluating marizomib’s IV formulation in an ongoing Phase II clinical trial in combination with dexamethasone in a highly refractory multiple myeloma population, including those refractory to carfilzomib.
The drug is also being evaluated in a Phase I/II study in in combination with pomalidomide and dexamethasone in relapsed and refractory multiple myeloma. Triphase is developing an oral formulation that is at present in IND-enabling studies.
Myeloma is a cancer caused by abnormal production of plasma cells which multiply uncontrollably and release only one type of antibody called paraprotein. Paraprotein has no known useful function. The disease does not exist as a tumor, but causes medical problems due to build-up of abnormal plasma cells in the bone marrow, as well as presence of paraprotein in the blood or urine.
The disease is also called multiple myeloma as it affects multiple places in the body including the skull, pelvis, and rib cage. Myeloma is a relapsing-remitting cancer and is the second most common form of bone marrow cancer.
Mr. Stonebacks will present clinical developments on marizomib as well as its new orphan drug designation at the Biocom fourth annual Global Life Science Partnering Conference.