Caliway Announced EMA Orphan Drug Designation Granted to CBL-514 for Dercum's Disease Treatment

- 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.

NEW TAIPEI CITY, Nov. 12, 2024 /PRNewswire/ -- 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.

About EMA Orphan Drug Designation

To support the development and evaluation of new treatments for rare diseases, the EMA grants orphan drug designation to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that the prevalence in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development.

Orphan drug designation qualifies sponsors for incentives, including 10-year marketing exclusivity to sponsors of approved orphan products, tax deductions on clinical trial expenses by the individual regulations of each EU member state, reductions in application fees, and eligibility for regulatory assistance and guidance from the EMA in the design of a drug development plan.

For more information regarding Orphan Drug Designation, please visit the EMA official website: https://www.ema.europa.eu/en/human-regulatory-overview/orphan-designation-overview  

About Dercum's Disease

Dercum's disease is a rare disorder characterized by the development of painful lipomas primarily located on the trunk region, and the extremities close to the trunk. The pain is chronic (>3 months), symmetrical, often disabling, and resistant to traditional analgesics.

The etiology of Dercum's disease remains unknown. There is no approved drug nor recommended standard treatment for Dercum's disease yet. The current treatment options, including surgical removal, liposuction, electrotherapy, and inflammation inhibitors, are symptomatic, and the efficacies are limited with numerous adverse events. The clinical need for Dercum's disease remains unmet.

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 of Dercum's disease treatment in 2030 is estimated to expand to $19.95 billion.

About CBL-514

CBL-514, a potentially first-in-class small-molecule drug, is a lipolysis injection that can induce adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity in treatment areas without causing any systematic side effects on the central nervous system, cardiovascular system, and respiratory system. Caliway's preclinical studies have shown that CBL-514 upregulates the apoptosis mediators caspase 3 and Bax/Bcl-2 ratio and then induces adipocyte apoptosis in vivo and in vitro.

Caliway is investigating multiple indications for CBL-514, including non-invasive subcutaneous fat reduction, Dercum's disease, and cellulite. To date, a total of 521 subjects have enrolled in CBL-514 clinical studies, with 317 subjects having received CBL-514 injection treatment. According to the results from 8 completed clinical studies of three indications, CBL-514 has demonstrated precise efficacy with a favorable safety profile. The global Pivotal Phase 3 study for non-surgical fat reduction is expected to be initiated in 2025.

About Caliway Biopharmaceuticals

Caliway Biopharmaceuticals (Caliway) is a Taiwan-headquartered, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics. Listed on the Taipei Exchange (TWSE-6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases. For more information, please visit: https://www.caliway.com.tw/en

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SOURCE Caliway Biopharmaceuticals