Taiwan's Caliway Biopharmaceuticals Receives EMA Orphan Drug Designation for CBL-514 in Dercum’s Disease Treatment
14 November 2024 | Thursday | News
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CBL-514 is the first and only drug to receive EMA Orphan Drug Designation for Dercum's disease treatment.
- CBL-514 received both FDA Orphan Drug Designation and Fast Track designation for Dercum's disease treatment earlier this year.
- CBL-514 Phase 2 study for Dercum's disease showed that it could significantly reduce lipoma dimension by 50%, and improve pain by 4.7 points (out of 10 points).
- The U.S. FDA-approved CBL-514 Phase 2b study is recruiting subjects; the study results are anticipated in Q4 2025.
Caliway Biopharmaceuticals (Caliway) announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to its novel small molecule drug, CBL-514, for the treatment of Dercum's disease. This designation will enable Caliway to access significant clinical development support, pre-authorization, and post-authorization activities fee reductions, and a ten-year market exclusivity upon approval within the EU.
Notably, CBL-514 is the first drug globally to receive dual designations from the U.S. Food and Drug Administration (FDA)—both Fast Track and Orphan Drug Designations—for Dercum's disease. With these recognitions from both the EMA and the FDA, Caliway anticipates these regulatory advantages will accelerate CBL-514's clinical development.
Caliway has completed a CBL-514 Phase 2 study (CBL-0201DD, NCT05387733) for Dercum's disease in 2023. The study results demonstrated that after administrating CBL-514 treatment, 64.5% of painful lipomas showed a dimension reduction of more than 50%, and 38.7% of painful lipomas showed complete clearance, with pain being reduced by 4.7 points.
Currently, there is a placebo-controlled Phase 2b study (CBL-0202DD, NCT06303570) evaluating CBL-514's efficacy in treating Dercum's disease undergoing. With subject recruitment initiated in August 2024, the study results are anticipated in Q4 2025.
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. This progress positions CBL-514 as a potential first-in-class therapy for Dercum's disease, offering new hope to patients who suffer from this rare disease worldwide.
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