April 24, 2025 brought important news to both researchers and patients gathered at the World Orphan Drug Congress in Boston. For those who follow clinical research, the rare condition known as Dercum’s disease has long been a stark reminder of how challenging unmet needs can be. Dercum’s disease is marked by the development of numerous painful fatty lumps under the skin, and until now, patients have faced persistent pain with no therapies officially approved. That changed when Caliway Biopharmaceuticals revealed new clinical trial results for their investigational therapy, CBL-514, which had just completed a phase 2 study aimed directly at this neglected disease.

CBL-514 is a small molecule administered by injection that targets the fatty tissue lipomas that cause pain in Dercum’s disease. Its journey began in the world of medical aesthetics but has shifted toward treating a very different type of patient—those who suffer daily from this rare disorder. In the clinical trial, researchers set out to gauge not only whether CBL-514 could shrink the painful lumps but also whether it could bring lasting relief from pain itself. The numbers were compelling: nearly two-thirds of the lipomas treated with CBL-514 shrank by at least half or disappeared altogether. Even more striking, more than half of the treated areas responded after just one round of therapy. Alongside the size reduction, patients reported an average pain decrease of nearly five points on a ten-point scale, results that can be life changing for someone dealing with chronic pain every day.

The study hit all of its primary and secondary goals, confirming the potential of CBL-514 to change how Dercum’s disease is managed. Not surprisingly, these results captured attention at the Congress. The gathering itself is a major annual event bringing together representatives from academia, regulatory bodies, biotech companies, patient groups, and investors. This year, there was added focus on drugs for orphan and rare diseases—fields that are often underfunded and overlooked but where new science and regulatory changes are now speeding things along.

CBL-514’s results do not just offer another experimental therapy—they show that progress is being made, and that clinical research can translate directly to a better quality of life for people who have gone far too long without effective treatment. After these phase 2 results, CBL-514 now moves into a larger, FDA-approved phase 2b trial. Recruitment is already underway, and the next set of results could come before the end of this year. Caliway’s drug is also uniquely positioned, having been awarded orphan drug status on both sides of the Atlantic, which provides regulatory and financial support as studies continue.

If the next trials are equally successful, CBL-514 may soon become the first dedicated treatment for Dercum’s disease, shifting how this rare disorder is managed worldwide. For patients and families who have waited so long, the sense of hope is tangible. The recognition of this work at one of the world’s leading rare disease gatherings was met with enthusiasm and cautious optimism. In the landscape of clinical research, moments like these remind us why the pursuit of new therapies matters so much. The story of CBL-514 will no doubt be watched closely in the months to come, as researchers, clinicians, and patients wait for the next chapter in a journey that might finally end with an approved treatment for this overlooked disease.