Caliway Biopharmaceuticals has announced that its investigational drug, CBL-514, has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Dercum's disease. This regulatory milestone provides Caliway with significant clinical development support, fee reductions, and a 10-year market exclusivity period in the EU upon approval.
CBL-514 is a novel small molecule drug and the first to receive both Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for Dercum's disease. These designations from both the EMA and FDA are expected to accelerate the clinical development of CBL-514.
Clinical Trial Results
A Phase 2 study (CBL-0201DD, NCT05387733) of CBL-514 for Dercum's disease was completed in 2023. Results showed that 64.5% of painful lipomas experienced a dimension reduction of more than 50% after CBL-514 treatment, and 38.7% of painful lipomas showed complete clearance. Patients also reported a 4.7-point reduction in pain.
Currently, a placebo-controlled Phase 2b study (CBL-0202DD, NCT06303570) is ongoing to further evaluate CBL-514's efficacy in treating Dercum's disease. Subject recruitment began in August 2024, and study results are anticipated in Q4 2025.
Mechanism of Action and Potential
CBL-514 induces adipocyte apoptosis, offering a novel approach to fat reduction. It is currently the only drug in advanced clinical stages demonstrating statistically significant efficacy results. This positions CBL-514 as a potential first-in-class therapy for Dercum's disease.
About Dercum's Disease
Dercum's disease is a rare disorder characterized by painful lipomas, primarily located on the trunk and extremities. The pain is chronic, symmetrical, and often resistant to traditional analgesics. The etiology of Dercum's disease remains unknown, and there is currently no approved drug or standard treatment. Current treatments, such as surgical removal and liposuction, offer limited efficacy and are associated with adverse events, highlighting the unmet clinical need.
Market Overview
The global Dercum's disease treatment market was valued at $11.3 billion in 2021 and is projected to reach $19.95 billion by 2030, with a compound annual growth rate (CAGR) of 6.76%.
About CBL-514
CBL-514 is a small-molecule lipolysis injection designed to induce adipocyte apoptosis and lipolysis, reducing subcutaneous adiposity without systemic side effects. Preclinical studies have demonstrated that CBL-514 upregulates apoptosis mediators, leading to adipocyte apoptosis in vivo and in vitro. Caliway is also investigating CBL-514 for non-invasive subcutaneous fat reduction and cellulite. To date, 521 subjects have been enrolled in CBL-514 clinical studies, with 317 receiving the injection. Results from eight completed clinical studies have shown precise efficacy and a favorable safety profile. A global Pivotal Phase 3 study for non-surgical fat reduction is expected to begin in 2025.
