The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller population sizes and are less understood. Drug companies tended to stick to drug development in better known and more profitable spheres. The Orphan Drug Act established incentives for drug companies whose therapies received Orphan Drug designation: fee waivers, tax credits, and up to seven years of market exclusivity if the drug is approved. 

Biotechnology company CellCentric recently shared that its product inobrodib was granted Orphan Drug designation for the treatment of multiple myeloma. Unfortunately, many people with multiple myeloma often relapse after treatment. The cancer also becomes more difficult to treat over time. Identifying novel solutions to improve patient lives drives companies like CellCentric. 

Learning about Inobrodib

To begin, let’s first go over what inobrodib is. Taken orally in a capsule, inobrodib is a first-in-class small molecule p300/CBP inhibitor. CellCentric explains that inobrodib impacts how MYC, IRF4, and the androgen receptor are expressed; these have all been found to be key cancer drivers. Outside of multiple myeloma, inobrodib is also being evaluated as a potential treatment for acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and non-Hodgkin’s lymphoma (NHL). Data from a study of inobrodib within these indications should be available by the end of the year.

CellCentric believes that inobrodib could potentially work as a monotherapy (especially in people whose condition is refractory to other treatments) or in conjunction with other treatments. 

The Facts: Multiple Myeloma

Multiple myeloma is a rare cancer that forms in plasma cells, a type of white blood cell. The plasma cells don’t function properly and begin to proliferate (grow) excessively. This crowds healthy blood cells out of the bone marrow, leading to a number of health issues. Multiple myeloma also causes abnormal antibodies called M proteins to be overproduced. If your doctor finds a high level of M proteins in your bloodwork, it signifies this cancer. Multiple myeloma is slightly more common in males than females, and typically occurs in people over 40 years old. Signs and symptoms vary from person-to-person; some people are more symptomatic while some show few symptoms. Potential signs of multiple myeloma may include:

• Recurrent infections
• Being more thirsty than usual
• Extremely pale skin
• Anemia (low red blood cell count)
• Weakness or numbness in the legs
• Fatigue or feeling more tired than usual
• Mental fogginess or confusion
• Nausea and vomiting
• Unintentional weight loss
• High calcium levels in the blood
• Appetite loss
• Bone pain in the spine or chest (though it can occur elsewhere)
• Constipation