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 upon approval in the EU.
CBL-514 is also 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 will accelerate CBL-514's clinical development.
Clinical Trial Results
A Phase 2 study (CBL-0201DD, NCT05387733) of CBL-514 for Dercum's disease, completed in 2023, demonstrated promising results. The study 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 pain reduction of 4.7 points on a 10-point scale.
Currently, a placebo-controlled Phase 2b study (CBL-0202DD, NCT06303570) is evaluating CBL-514's efficacy in treating Dercum's disease. Subject recruitment began in August 2024, and the study results are anticipated in Q4 2025.
About Dercum's Disease
Dercum's disease is a rare disorder characterized by the development of 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 recommended standard treatment. Existing treatments, such as surgical removal and liposuction, offer limited efficacy and are associated with adverse events.
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 potentially first-in-class small-molecule drug designed as a lipolysis injection to induce adipocyte apoptosis and lipolysis, reducing subcutaneous adiposity in treated areas. Preclinical studies have shown that CBL-514 upregulates apoptosis mediators, leading to adipocyte apoptosis in vivo and in vitro. Caliway is investigating CBL-514 for multiple indications, including non-invasive subcutaneous fat reduction, Dercum's disease, and cellulite. To date, 521 subjects have been enrolled in CBL-514 clinical studies, with 317 having received the injection. Results from eight completed clinical studies have demonstrated efficacy and a favorable safety profile. A global Pivotal Phase 3 study for non-surgical fat reduction is expected to begin in 2025.
Given its mechanism of action inducing adipocyte apoptosis, CBL-514 represents a potential first-in-class therapy for Dercum's disease.
