Camurus CAM2029 to treat polycystic liver disease granted orphan drug status by USFDA

Polycystic liver disease (PLD) is a rare, genetic, and chronic disorder characterized by progressive growth of cysts in the liver which can cause severe symptoms and result in an impaired quality of life of patients. There is today no approved pharmacological treatment available for PLD.

"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," says 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," he added.

Camurus has also recently received a Safe-to-Proceed letter from the FDA for the start of a randomized placebo-controlled Phase 2/3 study designed to assess efficacy and safety of CAM2029 in patients with symptomatic PLD.

Orphan drug designation qualifies the drug developer for a variety of development incentives and the prospect of seven years of market exclusivity, if approved. The FDA's ODD program is designed to advance the development of drugs for the treatment of rare diseases affecting fewer than 200.000 people in the US. 

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