CBL-514 is the first treatment to ever receive Orphan Drug Designation from the European Medicines Agency (EMA) for Dercum’s disease.
- In addition, CBL-514 was previously awarded Orphan Drug and Fast Track designations by the U.S. FDA for treating Dercum’s disease at the start of this year.
- In Phase 2 studies, CBL-514 demonstrated the ability to reduce lipoma (fatty tumor) volume by 50% and improve pain scores by 4.7 points out of a 10-point scale.
- A U.S. FDA-approved Phase 2b study for CBL-514 is currently recruiting participants, with results expected by the fourth quarter of 2025.
Caliway Biopharmaceuticals (Caliway) recently announced that the European Medicines Agency (EMA) has awarded Orphan Drug Designation (ODD) to their new small molecule medication, CBL-514, for treating Dercum’s disease. This designation is designed to support Caliway in its efforts to develop an effective therapy, providing regulatory assistance, fee reductions, and extended market exclusivity for up to ten years within the EU.
This recognition makes CBL-514 the first drug in the world to receive both Fast Track and Orphan Drug Designations from the U.S. FDA for Dercum’s disease, a milestone that Caliway hopes will enhance the global impact of CBL-514.
In 2023, Caliway initiated Phase 2 clinical trials (CBL-0201DD, NCT05387733) for CBL-514 to address Dercum’s disease. The initial results are promising: 64.5% of patients saw lipoma size reductions of over 50%, and 38.7% experienced the complete disappearance of certain lipomas, alongside an average pain score improvement of 4.7 points.
A placebo-controlled Phase 2b trial (CBL-0202DD, NCT06303570) is currently underway to evaluate how effective CBL-514 is in treating Dercum’s disease. Recruitment began in August 2024, and the results are anticipated by the fourth quarter of 2025.
Using an innovative approach to target fat cell apoptosis, CBL-514 stands as a unique and highly promising treatment option. This development positions CBL-514 as a groundbreaking potential solution for Dercum’s disease, providing renewed hope to patients who have faced limited treatment options worldwide.