FDA Denies Camurus' Hormonal Disorder Treatment Over Production Issues

The US Food and Drug Administration (FDA) has denied approval for Camurus’ new subcutaneous formulation of octreotide, named CAM2029, intended for treating acromegaly. This Swedish pharmaceutical company revealed that the FDA’s decision stemmed from identified deficiencies at a third-party manufacturing site.

Acromegaly, a rare hormonal disorder affecting approximately 60 in every million people, is characterized by abnormal bone growth and enlarged hands, feet, and facial features. It often results from a tumor in the pituitary gland that causes an overproduction of growth hormone and insulin-like growth factor-1. This condition can impair the quality of life and increase mortality risk if left unchecked. Common symptoms include headaches, joint pain, and fatigue.

CAM2029 is an extended-release subcutaneous formulation of octreotide, designed to provide a more convenient once-monthly dosing schedule for patients. Octreotide is a peptide drug that mimics somatostatin, a natural hormone, to inhibit the secretion of growth hormone, thereby addressing acromegaly's underlying cause. This drug also helps reduce insulin and glucagon levels and activity.

In a Complete Response Letter, the FDA pointed out facility-related deficiencies found during a recent inspection of Camurus’ third-party manufacturing partner. Importantly, the letter did not raise concerns about the clinical efficacy or safety of CAM2029. According to the company, the FDA requires satisfactory responses from the third-party provider to address these deficiencies to move forward.

Fredrik Tiberg, CEO of Camurus, expressed disappointment over the CRL but remained optimistic about the drug's potential and the supporting data. He emphasized the company's commitment to working with their external manufacturer and the FDA to make CAM2029 available to acromegaly patients as swiftly as possible. Tiberg noted that discussions with the FDA regarding CAM2029’s labeling are in advanced stages.

The rise in FDA rejections due to manufacturing issues is notable. From 2017 to 2023, 30% of the FDA's 238 Complete Response Letters were due to problems related to manufacturing processes, facility inspections, or chemical, manufacturing and controls.

Camurus has highlighted the need to address the FDA’s concerns expediently, aiming to offer an improved treatment option for those living with acromegaly. Unlike the current standard requiring multiple daily doses, CAM2029 promises better biochemical control and enhanced quality of life with its monthly dosage regimen.

Octreotide has established itself as an effective treatment for acromegaly over the years. Sun Pharmaceuticals received FDA approval in 1988 for its octreotide acetate formulation, marketed as Bynfezia Pen, for three-times-daily subcutaneous treatment. Besides acromegaly, Bynfezia Pen is also approved for managing carcinoid tumors and vasoactive intestinal peptide tumors.

The development of CAM2029 marks a significant step towards providing acromegaly patients with more convenient treatment options. The extended-release formulation has shown promising results in the Phase III ACROINNOVA 2 study, indicating better control of acromegaly symptoms and improvement in patients' overall well-being compared to the existing standard of care.

Camurus’ journey to bring CAM2029 to market continues as they address the FDA's requirements, focusing on resolving the manufacturing setbacks. The company remains committed to meeting the unmet medical needs of acromegaly patients, providing hope for more effective and manageable treatment solutions in the near future.