Drug development is important in creating new therapeutic options for patients with rare or chronic illnesses. One incentivizing program is the Orphan Drug program. Orphan Drug designation is granted to drugs and biologics intending to treat, diagnose, or prevent rare diseases, defined as impacting under 200,000 U.S. citizens. Once a drug receives this status, the drug developer also receives a variety of benefits, such as fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. According to a news release from biopharmaceutical company C4 Therapeutics (“C4”), the company’s therapy CFT7455 recently received Orphan Drug designation for the treatment of multiple myeloma (MM). 

CFT7455

The Orphan Drug designation follows a Phase 1/2 clinical trial to evaluate the safety, efficacy, tolerability, pharmacokinetics, and pharmacodynamics of CFT7455. Currently, the trial is still enrolling. Altogether, an estimated 160 patients will enroll. Researchers hope to understand what type of anti-tumor response CFT7455 prompts in patients with MM and other forms of non-Hodgkin’s lymphoma (NHL). If this MonoDAC degrader, which targets IKZF1/3, is effective, it could signal a new line of treatment – and potentially better outcomes – for patients.

Multiple Myeloma (MM)

Multiple myeloma (MM) is a rare cancer which forms in plasma cells, a type of white blood cells. While doctors are not exactly sure what causes MM, some believe that chromosome 13 abnormalities could play a role. Normally, plasma cells fight infections by creating antibodies which fight foreign invaders. But in patients with MM, abnormal cancer cells create abnormal M proteins, crowding out healthy blood cells and antibodies. Risk factors for developing MM include being male, being Black, older age (60+), or having a family history of multiple myeloma or other cancers. Symptoms associated with MM include:

• Nausea and vomiting
• Bone pain, particularly in the spine and chest
• Constipation
• Fever
• Excessive thirst and frequent urination
• Appetite loss
• Frequent infections
• Weakness or numbness in the legs
• Hypercalcemia (too much calcium in the blood)
• Fatigue
• Unintended weight loss
• Mental fogginess or confusion
• Reduced kidney function and/or kidney failure
• Anemia (low red blood cell count)