Caliway Biopharmaceuticals has announced that its novel small molecule drug, CBL-514, has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Dercum's disease. This regulatory milestone follows a similar designation and Fast Track status granted by the U.S. Food and Drug Administration (FDA), potentially accelerating the drug's clinical development for this rare and debilitating condition.
Dercum's disease is characterized by the development of painful lipomas, primarily on the trunk and extremities. The pain is chronic, often disabling, and resistant to traditional analgesics. Currently, there are no approved drugs or recommended standard treatments, with existing options limited to symptomatic relief through surgical removal, liposuction, electrotherapy, and inflammation inhibitors.
Clinical Efficacy of CBL-514
CBL-514 is a lipolysis injection designed to induce adipocyte apoptosis and lipolysis, reducing subcutaneous adiposity in treated areas. A Phase 2 study (CBL-0201DD, NCT05387733) completed in 2023 demonstrated promising results. According to Caliway, 64.5% of painful lipomas showed a dimension reduction of more than 50% after CBL-514 treatment, and 38.7% of painful lipomas showed complete clearance, with pain being reduced by 4.7 points.
"Given its innovative approach to fat reduction through adipocyte apoptosis, CBL-514 is the only drug in advanced clinical stages with statistically significant efficacy results," the company stated. This progress positions CBL-514 as a potential first-in-class therapy for Dercum's disease.
Ongoing Clinical Trials
A placebo-controlled Phase 2b study (CBL-0202DD) is currently underway to further evaluate CBL-514's efficacy in treating Dercum's disease. Subject recruitment began in August 2024, with results anticipated in Q4 2025.
Market and Regulatory Advantages
The EMA's Orphan Drug Designation provides Caliway with several incentives, including reduced fees for clinical development, pre-authorization, and post-authorization activities, as well as a ten-year market exclusivity upon approval within the EU. 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, reflecting a compound annual growth rate (CAGR) of 6.76%.
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 treatment. According to results from eight completed clinical studies across three indications, CBL-514 has demonstrated precise efficacy with a favorable safety profile. A global pivotal Phase 3 study for non-surgical fat reduction is expected to begin in 2025.
