New Myeloma Treatment Gets Orphan Drug Designation

The FDA granted OM-301 an Orphan Drug Designation for patients with myeloma.

The Food and Drug Administration (FDA) granted OM-301 an Orphan Drug Designation for the treatment of patients with multiple myeloma, according to an April 11 announcement from Oncolyze, the pharmaceutical company manufacturing the agent.

Therapies are given Orphan Drug Designation when they have the potential to prevent, diagnose or treat a rare disease or condition — such as myeloma, which has approximately 35,000 cases diagnosed each year — according to the agency’s website.

Through the designation, the FDA offers tax credits for qualified clinical trials, exemptions of certain fees and potential market exclusivity for seven years to the pharmaceutical company, to promote drug manufacturers to put a focus on developing drugs for rarer diseases.

In vitro research (meaning that it took place outside a human body) showed that OM-301 was effective in fighting eight different multiple myeloma cell lines, including p53-mutant and null cell lines. Early research showed that it prolonged survival.

Now, Oncolyze plans on initiating clinical trials where OM-301 is administered intravenously to patients with myeloma.

READ MORE: Onward in the Fight Against Multiple Myeloma

OM-301 works by binding to HDM2, a protein found on the surface of myeloma cells, then binding to an external surface on the cell membrane and killing the cancer. By targeting the cancer-specific protein, OM-301 is able to destroy myeloma while sparing surrounding healthy cells.

Of note, OM-301 already has an Orphan Drug Designation for the treatment of patients with acute myeloid leukemia, granted by the FDA in January 2022.

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