Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer than 200,000 people. This can make the drug development process slow and difficult; many companies struggle to create therapies for rare diseases as the populations are often smaller and drug development isn’t as profitable. The Orphan Drug designation incentivizes this process, creating a more equitable drug development sphere that is inclusionary of those with rare conditions. For example, companies whose products receive this status earn fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is accepted. Pharmaceutical Technology recently reported that the FDA granted Orphan Drug designation to ISB 2001, a treatment for people living with multiple myeloma.

ISB 2001 is a first-in-class trispecific antibody that targets CD3, BCMA, and CD38. CD3 is expressed on T-cells and BCMA and CD38 on multiple myeloma cells. In a presentation at the American Society of Hematology’s Annual Meeting, which took place in December 2022, drug developer Ichnos Sciences shared that ISB 2001 showed high potency, superior cytotoxicity compared to other therapies, and efficacy in causing tumor cell death.

Moving forward, Ichnos Sciences plans to launch a Phase 1 dose escalation study by the end of this year.

About Multiple Myeloma (MM)

Multiple myeloma is a rare blood cancer that affects plasma cells, a type of white blood cell that normally fight infections and create antibodies. This cancer forms when normal cells become abnormal and cancerous. These abnormal cells then begin producing abnormal antibodies called M proteins. Abnormal cells crowd healthy blood cells out of the bone marrow, leading to additional health problems. Multiple myeloma is more common in females, people between 40-70 years old, and Black individuals. There are no cures for this cancer. A treatment plan may encompass numerous therapies such as immunomodulatory drugs, chemotherapy, and HDAC inhibitors (among others). Symptoms of multiple myeloma may, but do not always, include:

• Bone pain, often in the chest or spine
• Weakness or numbness in the arms and legs
• Nausea and vomiting
• Unintentional weight loss
• Anemia (low red blood cell count)
• Extreme fatigue and weakness
• Shortness of breath
• Sudden bone fracturing without a known cause
• Excessive thirst
• Unexplained fever
• Appetite loss
• Constipation
• Mental fogginess or confusion
• Easy bruising or bleeding
• Frequent or recurrent infections
• Hypercalcemia (high blood calcium levels)