Have you ever heard of the Orphan Drug Act? This Act helped stimulate and advance the development of, and research into, therapeutics for individuals living with rare or underserved conditions. In the United States, “rare” conditions are those affecting 200,000 people or fewer. Prior to the passage of the Orphan Drug Act, it was difficult to drive commercial investment into research due to the small population sizes of some rare conditions. However, the law offered incentives for this type of drug development through the Orphan Drug designation. Those whose therapies received Orphan Drug designation also earned tax credits, fee waivers, increased regulatory communication with the FDA, and seven years of market exclusivity upon drug approval. According to an April 14, 2023 article in CURE, OM-301 recently earned Orphan Drug designation for the treatment of multiple myeloma.

Developed by pharmaceutical company Oncolyze, OM-301 is an investigational therapy, delivered intravenously, that binds to HDM2. HDM2, which regulates tumor suppressor protein p53, can be found on the surface of some cancer cells. In 2022, OM-301 also received Orphan Drug designation for the treatment of acute myeloid leukemia (AML). When we discussed that on Patient Worthy, we also shared how the drug works: by creating pores, or holes, in cancer cells, causing apoptosis (cellular death).

So far, preclinical studies of OM-301 have shown that it is effective in treating eight separate cell lines related to multiple myeloma. Further, the treatment seemed to improve survival rates. Moving forward, Oncolyze hopes to begin in-human studies to continue evaluating the drug.

About Multiple Myeloma

In our body, we have plasma cells (a type of white blood cell) that form in the bone marrow. These plasma cells play a role in immune response and how the body protects against “foreign invaders,” like viruses or germs. But in people with multiple myeloma, cancer begins in these plasma cells. These cancerous cells then act and proliferate abnormally, crowding healthy cells out of the bone marrow. If only one tumor forms, it is called solitary plasmocytoma. If multiple tumors form, or the bone marrow contains over 10% abnormal plasma cells, it is considered multiple myeloma. The exact cause of multiple myeloma is unknown, though doctors have noted issues with chromosome 13 (partially or fully missing) in myeloma cells. Multiple myeloma is slightly more common in men than women; it is also significantly more common in older individuals.

Below, you will find some symptoms of multiple myeloma. It’s important to note, though, that not every person will experience all of these symptoms or symptoms of the same severity. The below is also not an exhaustive list of symptoms:

• Bone pain, often in the chest, ribs, spine, or lower back
• Hypercalcemia (high blood calcium levels)
• Unintentional weight loss
• Appetite loss
• Nausea
• Excessive thirst
• Shortness of breath
• Thrombocytopenia (low platelet count)
• Anemia (low red blood cell count)
• Pallor (extremely pale skin)
• Fatigue
• Muscle pain and weakness
• Constipation
• Mental fogginess or confusion
• Frequent or recurrent infections
• Weakness or numbness in the lower extremities

There are a number of treatment options available for people living with this condition. You can find a full list of approved treatments here.