The FDA has issued a Complete Response Letter (CRL) to Camurus regarding its New Drug Application (NDA) for CAM2029 (octreotide) extended-release injection, a treatment for acromegaly. The decision, announced by Camurus on October 22, 2024, stems from deficiencies discovered during a recent Current Good Manufacturing Practices (cGMP) inspection of a third-party manufacturing facility.
According to the FDA, the approval of the NDA is contingent upon satisfactory responses from the third-party manufacturer regarding the inspection findings. The agency has clarified that the CRL does not raise concerns about the clinical efficacy or safety of CAM2029.
Company Response and Next Steps
Fredrik Tiberg, President & CEO of Camurus, expressed disappointment but reaffirmed confidence in the drug's potential. "The CRL is disappointing; however, we are confident in the data supporting our NDA and the potential of CAM2029 to address unmet medical needs of patients with acromegaly," Tiberg stated. Camurus is committed to working with the FDA and the third-party manufacturer to address the issues and expedite the drug's availability to patients.
Labeling discussions with the FDA were reportedly advanced during the NDA review, suggesting progress in other aspects of the application.
CAM2029: A Novel Octreotide Formulation
CAM2029 is an investigational, ready-to-use octreotide subcutaneous depot designed for monthly administration via a prefilled pen. It is being developed for acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD). Clinical trials have demonstrated approximately five-fold higher bioavailability compared to existing long-acting intramuscular octreotide formulations.
The CAM2029 clinical program for acromegaly included seven clinical trials, featuring the ACROINNOVA program. Phase 3 data demonstrated superior biochemical control compared to placebo, along with improvements in symptom control, treatment satisfaction, and quality of life compared to standard-of-care somatostatin receptor ligands (SRLs) like octreotide and lanreotide. The safety profile was consistent with existing treatments.
Acromegaly: An Unmet Need
Acromegaly is a rare, slowly progressive disorder caused by excess growth hormone (GH) production, often due to pituitary tumors. This leads to elevated insulin-like growth factor-1 (IGF-1) levels, resulting in abnormal growth of bone and tissue. Symptoms include enlarged hands, feet, facial features, fatigue, joint pain, and headaches. The prevalence is estimated at 60 cases per million.
Broader Implications
While the US NDA faces delays, the Market Authorization Application for CAM2029 in the EU remains under review. Development programs for GEP-NET and PLD are also ongoing and unaffected by the CRL. This regulatory setback highlights the critical importance of manufacturing quality and compliance in pharmaceutical development and approval processes.
