Polycystic liver disease is a rare genetic disorder characterized by the progressive growth of more than 10 cysts in the liver. The disorder may cause rare complications including hepatic cyst hemorrhage, infection, or rupture.
CAM2029 is an investigational, ready-to-use, long-acting octreotide subcutaneous injection depot. CAM2029 is designed using the Company’s proprietary FluidCrystal® injection depot technology to provide fast onset and 1 month duration. The investigational treatment has also shown high bioavailability, including a 500% higher bioavailability compared with Sandostatin LAR.
The Company plans to initiate a phase 2/3 study evaluating the efficacy and safety of CAM2029 in patients with symptomatic polycystic liver disease.
“The orphan drug designation for CAM2029 is a significant milestone in our efforts to develop an effective pharmacological treatment for people with polycystic liver disease and a recognition of the importance of our overall development program for CAM2029,” said Dr Fredrik Tiberg, CEO and Head of R&D at Camurus. “CAM2029 is designed to address a significant unmet medical need in this population and could potentially become the first approved pharmacological treatment for PLD in the US market.”
The Company is also investigating CAM2029 for the treatment of acromegaly and gastroenteropancreatic neuroendocrine tumors.
- FDA grants Orphan Drug designation in the US for CAM2029 for the treatment of polycystic liver disease. News release. Camurus. Accessed September 16, 2021. https://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-in-the-us-for-cam2029-for-the-treatment-of-polycystic-liver-disease-301378447.html.
- Pipeline. Camurus. Accessed September 16, 2021. https://www.camurus.com/pipeline/.
This article originally appeared on MPR