Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, diagnose, or prevent a rare disease or condition. “Rare,” in the United States, is defined as any condition affecting fewer than 200,000 Americans. The Orphan Drug designation was created through the Orphan Drug Act to incentivize drug development within the rare disease space. As a result, those whose products receive this designation are also granted increased regulatory assistance, fee waivers, tax credits, and seven years of market exclusivity upon approval. According to a January 5, 2023 news release, the FDA recently granted Orphan Drug designation to LP-284 for mantle cell lymphoma (MCL).
Developed by biopharmaceutical company Lantern Pharma, Inc., LP-284 is a novel small molecule agent. It works to damage cancer cell DNA that has specific mutations. Outside of MCL, LP-284 is being explored as a potential therapeutic option for double hit lymphoma. So far, preclinical studies have shown that LP-284 offers anti-tumor properties, including in previously treatment-resistant tumors. Given that so many patients with MCL tend to relapse following treatment, finding new treatment options is necessary. LP-284 has the potential to fill that need.
What is Mantle Cell Lymphoma (MCL)?
Mantle cell lymphoma is a B-cell lymphoma that develops from cancerous white blood cells called lymphocytes within the mantle zone of the lymph nodes. This cancer exists under the greater umbrella of non-Hodgkin’s lymphoma (NHL). Doctors are unsure of the exact cause of mantle cell lymphoma. However, many theorize that MCL results from a gene mutation that triggers the release of cyclin D1, a protein which causes uncontrolled B-cell growth. While MCL often progresses slowly at the onset of the disease, it becomes more aggressive over time.
Unfortunately, it can be difficult to identify MCL in early stages of the disease. Many people are asymptomatic. As symptoms appear, they can include:
- Swollen lymph nodes in the neck, throat, elbows, shoulders, and chest
- Appetite loss
- Heartburn and indigestion
- Enlarged liver, spleen, and/or tonsils
- Nausea and vomiting
- Unexplained itching
- Drenching night sweats
- Lower back pain and/or pressure that often continues down one or both legs
- Diarrhea or other gastrointestinal issues
- Repeated fever with no known cause
- Unintended weight loss
- Headache, dizziness, or poor balance
- Confusion or irritability
Treatment options can include chemotherapy, immunotherapy, targeted therapy, and stem cell transplantation.