CAM2029 Drug Granted Orphan Drug Designation To Treat Polycystic Liver Disease

Release Date: 05-Oct-2021

US FDA has granted orphan drug designation (ODD) for the company's investigational medicinal product, octreotide subcutaneous depot (CAM2029), for the treatment of autosomal dominant polycystic liver disease.


CAM2029 is a ready-to-use, long-acting subcutaneous depot of octreotide under development for treatment of three rare diseases; acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD). This drug developed for improved octreotide exposure and easy self-administration by patients using a prefilled pen injector. This drug has also been granted orphan designation in the EU for the treatment of acromegaly and in the US for the treatment of PLD.


The FDA Orphan Drug Designation Program gives orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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