The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for CAM2029 (octreotide) extended-release injection, intended for the treatment of acromegaly. The decision follows a Current Good Manufacturing Practices (cGMP) inspection of a third-party manufacturing facility in September 2024, which revealed facility-related deficiencies.
The FDA has communicated that resolution of these deficiencies by the third-party manufacturer is necessary before the NDA can be approved. According to Camurus, the CRL does not raise any concerns regarding the clinical efficacy or safety of CAM2029.
Fredrik Tiberg, Camurus' President & CEO, expressed disappointment but affirmed confidence in the data supporting the NDA and CAM2029's potential to address unmet needs in acromegaly treatment. Camurus is committed to working with the FDA and the third-party manufacturer to resolve the issues and bring CAM2029 to patients as quickly as possible.
Ongoing Regulatory and Development Activities
While the U.S. NDA is under review, a Market Authorization Application for CAM2029 for the treatment of acromegaly is also being evaluated in the European Union. Furthermore, Camurus is pursuing two additional development programs for CAM2029, targeting gastroenteropancreatic neuroendocrine tumors and polycystic liver disease. The CRL is not expected to impact the progress of these programs.
About Acromegaly and CAM2029
Acromegaly is a rare, slowly progressive endocrine disorder typically caused by a pituitary tumor that leads to excess growth hormone production and elevated insulin-like growth factor-1 (IGF-1) levels. This results in abnormal growth of bone and tissue, enlarged extremities and facial features, and various systemic symptoms, including fatigue, joint pain, headaches, and visual disturbances. The prevalence of acromegaly is estimated to be approximately 60 cases per million people. Inadequate control of biochemical markers and symptoms can significantly impair patients' quality of life and increase mortality.
CAM2029 is an investigational, ready-to-use octreotide subcutaneous depot formulation designed for enhanced octreotide exposure and convenient once-monthly administration. It is administered via a prefilled pen, facilitating self-administration by patients. Clinical trials have demonstrated that CAM2029 provides superior biochemical control compared to placebo and improvements in symptom management, treatment satisfaction, and overall quality of life. In Phase 3 trials, CAM2029 showed superior biochemical control compared to placebo as well as improvements in symptom control, treatment satisfaction, and quality of life compared to SoC at baseline with first-generation somatostatin receptor ligands (SRLs), octreotide and lanreotide. The safety profile of CAM2029 was consistent with SoC with no new findings.
