US FDA Grants Orphan Drug Designation (ODD) for CAM2029 to Treat Polycystic Liver Disease


The company ‘Camurus’ announced, the US FDA has granted the orphan drug designation (ODD) for the investigational medicinal product of the company known as octreotide subcutaneous depot (CAM2029), used for the treatment of the autosomal dominant polycystic liver disease in the people.

Dr. Fredrik Tiberg, CEO and head of Research and Development at Camurus said, the orphan drug designation (ODD) for the CAM2029 has been a significant milestone in the efforts of the company for developing an effective pharmacological treatment for the people suffering from the polycystic liver disease and it has also been a recognition of importance of the company’s overall development program for CAM2029.

Dr. Fredrik Tiberg also said, the CAM2029 has been designed for the purpose of addressing a significant medical need, which is not met in the current population, and it can potentially become the very first approve pharmacological treatment available for the polycystic liver disease in the market of the United States.

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The company ‘Camurus’ has also recently received a letter of Safe-to-Proceed from the US FDA for the purpose of starting a randomized phase 2 and phase 3 study controlled by placebo, which has been designed for assessing the safety and efficacy of the CAM2029 treatment in the patients with systematic polycystic liver disease.

The orphan drug designation (ODD) program of US FDA has been designed to advance in the development of the drugs for the treatment of the rare diseases that have been affecting lesser than 200,000 people in the United States of America.