OM-301 for AML Granted Orphan Drug Designation


The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, “rare” conditions are those affecting 200,000 or less Americans. According to a news release from January 5, 2022, OM-301, a therapy for acute myeloid leukemia (AML), recently earned this status.


To begin, what exactly is OM-301? Developed by disruptive cancer therapeutics and biotech company Oncolyze, Inc. (“Oncolyze”), OM-301 is described as:

an investigational drug candidate…delivered to patients as an intravenous infusion (potentially over a one-hour period) once daily. OM-301 is a fusion peptide that binds to HDM2 located on the cell surface of cancer cells and selectively creates pores in those cells, thereby inducing cell death.

Currently, Oncolyze is developing OM-301 as a potential treatment not just for AML, but for other cancer indications as well. These include multiple myeloma (MM) and solid tumors.

The Orphan Drug designation also comes with a variety of benefits for the drug developer (in this case, Oncolyze). These benefits include fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval.

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a cancer which often begins in the bone marrow and spreads to the blood. When damage occurs to DNA in developing cells within the bone marrow, the bone marrow begins producing immature and abnormal cells. These may be abnormal red blood cells, platelets, or myeloblasts (white blood cells). As these abnormal cells accumulate, it crowds out healthy blood cells. Ultimately, this makes it difficult for blood to work properly. Risk factors include older age, being male, prior radiation or chemical exposure, smoking cigarettes, or having certain genetic disorders. Symptoms associated with AML include:

  • Fever
  • Fatigue or general malaise
  • Shortness of breath and/or difficulty breathing
  • Pallor (extremely pale skin)
  • Easy bruising and bleeding
  • Frequent or recurrent infections
  • Pancytopenia
  • Bone pain
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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