Caliway Biopharmaceuticals announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to CBL-514, a novel small molecule drug, for the treatment of Dercum's disease. This designation provides Caliway with significant clinical development support, fee reductions, and a ten-year market exclusivity period upon approval in the EU.
Notably, CBL-514 is the first drug globally to receive both Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for Dercum's disease. Caliway anticipates that these regulatory advantages from both the EMA and FDA will accelerate CBL-514's clinical development.
Clinical Trial Results
A Phase 2 study (CBL-0201DD, NCT05387733) for Dercum's disease, completed in 2023, demonstrated that CBL-514 treatment resulted in a dimension reduction of more than 50% in 64.5% of painful lipomas, with 38.7% showing complete clearance. Patients also experienced a pain reduction of 4.7 points on a 10-point scale.
Currently, a placebo-controlled Phase 2b study (CBL-0202DD, NCT06303570) is underway 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.
About Dercum's Disease and CBL-514
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. There is currently no approved drug or recommended standard treatment for Dercum's disease, with existing treatments offering limited efficacy and numerous adverse events.
CBL-514 is a potentially first-in-class small-molecule drug designed as a lipolysis injection that induces adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity in treatment areas. Preclinical studies have shown 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.
Market Opportunity
According to the Global Dercum's Disease Market Research Report, the global Dercum's disease treatment market size in 2021 was $11.3 billion. With a compound annual growth rate (CAGR) of 6.76%, the global market is projected to reach $19.95 billion by 2030.
EMA Orphan Drug Designation Details
The EMA grants orphan drug designation to support the development of treatments for rare diseases with a prevalence of no more than 5 in 10,000 in the EU. The designation provides incentives, including 10-year market exclusivity, tax deductions on clinical trial expenses, reduced application fees, and regulatory assistance from the EMA.
