FDA Grants Orphan Drug Designation for CBL-514 to Treat Adult Patients With Dercum Disease


This designation makes CBL-514 the first drug to receive both Orphan Drug Designation and Fast Track Designation for the treatment of Dercum disease.

About the Trial

Trial Name: A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas

ClinicalTrials.gov ID: NCT05387733

Sponsor: Caliway Biopharmaceuticals Co., Ltd.

Completion Date: April 3, 2023.

Health care professional preparing injection -- Image credit: Alernon77 | stock.adobe.com

Image credit: Alernon77 | stock.adobe.com

The FDA has granted Orphan Drug Designation (ODD) to CBL-514 to treat Dercum disease. With this ODD, CBL-514 is the first drug to receive an ODD and Fast Track designation for the treatment of Dercum disease.1

Dercum disease is a rare disorder characterized by the development of painful lipomas—primarily located on the trunk region and extremities close to the trunk—with chronic pain that is symmetrical. These lipomas alongside chronic pain can cause significant challenges for patients; additionally, lipomas can be unresponsive to traditional analgesics. CBL-514 is a lipolysis injection that reduces local subcutaneous fat by inducing adipocyte apoptosis. It is a first-in-class small-molecule drug that has been injected in treatment areas and has demonstrated no systemic adverse effects (AEs) on the central nervous system, cardiovascular system, and respiratory system in studies that examined animals.

Nonclinical studies that evaluated CBL-514 showed that the drug upregulates the apoptosis mediators caspase 3 and Bax/Bcl-2 ratio, as well as induces dose-dependent adipocyte apoptosis both in vivo and in vitro. In addition to Dercum disease, CBL-514 is currently being investigated for multiple indications including non-invasive subcutaneous fat reduction, cellulite, and lipoma.

A phase 2 study (NCT05387733), which evaluated the safety and efficacy of CBL-514 in treating Dercum disease, enrolled participants aged 18 to 64 years who had at least 4 painful lipomas with a diameter of 1.0 cm to 6.0 cm as determined by an ultrasound. In addition, patients had to have chronic pain relating to Dercum disease for at least 3 months to be eligible for enrollment. After CBL-514 administration, injection volume per lipoma was then based on lipoma size which was determined by an ultrasound.

Each patient had up to 8 lipomas treated, with eligible participants randomly assigned to 2 dose groups, receiving either 10-mg or 15-mg injections. The number of injections was determined by the lipoma diameter and was consistent across both groups, with 1.0 cm to 2.0 cm receiving 1 injection; 2.0 cm to 3.5 cm receiving 2; 3.5 cm to 5.0 cm receiving 3; and 5.0 cm to 6.0 cm receiving 5. Injections were administered into selected lipomas.

The primary end point was the efficacy of CBL-514 as determined by the change of lipoma dimensions after 84 days. Secondary end points included changes in pain as assessed by the comparative pain scale, safety (as determined by AEs), and the incidence of clinically significant and abnormal findings after treatment. The study results demonstrated that following treatment with CBL-514, approximately 64.5% of painful lipomas showed dimension reduction of more that 50%. In addition, about 38.7% of painful lipomas demonstrated complete clearance, with patients’ pain being reduced by 4.7 on the comparative pain scale.

"Receiving Orphan Drug Designation and Fast Track Designation from the FDA is a huge milestone in CBL-514 development. Moreover, of all Dercum's disease treatment studies, CBL-514 is by far the most advanced program with the clinical efficacy being proven to reduce the size of the painful lipoma by more than 50% significantly," said Vivian Ling, CEO of Caliway Biopharmaceuticals, in a press release. "With these 2 designations, we look forward to accelerating CBL-514 clinical development, and becoming the first approved drug for Dercum's disease treatment with a 7-year marketing exclusivity."1

  1. Caliway Biopharmaceuticals. Caliway Announced Orphan Drug Designation Granted to CBL-514 for the Treatment of Dercum's Disease. News release. March 4, 2024. Accessed March 5, 2024.
  2. A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas.ClinicalTrials.gov identifier: NCT05387733. Updated April 20, 2023. Accessed March 5, 2024. https://clinicaltrials.gov/study/NCT05387733
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