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

Written by Ruchika Sharma Published On 2021-09-19T10:00:06+05:30 | Updated On 19 Sept 2021 10:00 AM IST

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

Sweden: Camurus has recently announced that the US Food and Drug Administration (USFDA) 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.

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.

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). CAM2029 has been successfully evaluated in four completed clinical Phase 1 and 2 studies and is being assessed in two ongoing pivotal Phase 3 studies in acromegaly and a pivotal Phase 3 study in GEP-NET. A Phase 2/3 study in patients with PLD is under preparation.

CAM2029 is developed for enhanced octreotide exposure and easy self-administration by patients using a prefilled pen injector. CAM2029 has been granted orphan designation in the EU for the treatment of acromegaly and in the US for the treatment of PLD.

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.

camuruscamurus newsusfdapolycystic liver diseaseoctreotide subcutaneous depot

Ruchika Sharma

Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751