In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening diseases or conditions. Rare conditions are those affecting fewer than 200,000 individuals. As a benefit of this designation, drug developers whose products receive Orphan Drug designation earn a variety of incentives, such as fee waivers, tax credits, and seven years of market exclusivity upon approval. According to an article in Myeloma Research News, QXL138AM, a treatment for multiple myeloma (MM), recently received Orphan Drug designation from the FDA.

What is QXL138AM?

Developed by immunotherapy company Nammi Therapeutics, QXL138AM is a masked immunocytokine (MIC) in which an:

anti-CD138 antibody is fused with IFNα2 at the C-terminus of the Ab heavy chains, a peptide mask that binds the IFNα2 and competes with its interaction with receptor is attached to the C-terminal end of the interferon by a linker that is cleavable by proteases enriched in the tumor microenvironment.

In short, QXL138Am promotes interferon-alpha (IFNα2) signaling in tumor cells. This allows the treatment to target and destroy cancer cells without any additional toxicity sometimes associated with other therapeutic options. The treatment uses an anti-CD138 antibody because CD138, a protein, is often more highly expressed on myeloma cells, making it a more targeted treatment option. 

Preclinical studies showed that QXL138AM offered durable and sustained responses in the body. Moving forward, Nammi Therapeutics hopes to have the opportunity to evaluate the therapy within clinical studies.

About Multiple Myeloma (MM)

Doctors are not sure exactly what causes multiple myeloma (MM), a rare form of cancer that forms in plasma cells, a type of white blood cell. However, partially or fully missing chromosome 13 could play a role. Normally, plasma cells help the body fight infection. But in multiple myeloma, abnormal cells crowd healthy cells out of the bone marrow, causing a number of issues. Symptoms of multiple myeloma can include:

• Nausea
• Unintended weight loss
• Appetite loss
• Bone pain, particularly in the spine, back, hips, chest, or skull
• Mental fogginess or confusion
• Excessive thirst
• Hypercalcemia (high blood calcium levels) 
• Blood hyperviscosity 
• Constipation
• Fatigue
• Anemia, leukopenia, and/or thrombocytopenia
• Frequent or recurrent infections
• Bone weakness