The regulator cited deficiencies at a third-party manufacturing facility. Camurus is seeking approval for its extended-release subcutaneous formulation of octreotide, which would allow more convenient once-monthly dosing for patients with acromegaly.
The FDA has rejected Camurus’ investigational subcutaneous formulation of octreotide, dubbed CAM2029, for the treatment of the rare hormonal disorder acromegaly, the Swedish drugmaker announced on Tuesday.
In a Complete Response Letter (CRL), the regulator cited “facility-related deficiencies” discovered last month during an inspection of a third-party manufacturer as the reason for the rejection and did not point to other issues, particularly as regards CAM2029’s clinical efficacy or safety data. The FDA needs to receive “satisfactory” responses from Camurus’ third-party provider before it can resolve the CRL, according to the company.
CEO Fredrik Tiberg in a statement called the CRL “disappointing” but noted that the company remains “confident in the data supporting our [New Drug Application] and the potential of CAM2029 to address unmet medical needs of patients with acromegaly.”
CRLs from the FDA rejecting drug candidates due to manufacturing issues are on the rise. Out of a total of 238 CRLs issued between 2017 and 2023, 30% involved quality/manufacturing issues—particularly problems with manufacturing processes, facility inspections, or chemical, manufacturing and controls.
Camurus will work with its external manufacturer and with the FDA “to bring CAM2029 to patients living with acromegaly as soon as possible,” Tiberg said. To this end, the company said that it has already held labelling discussions with the regulator and that CAM2029’s prescribing information is “well advanced.”
Afflicting around sixty patients per one million people, acromegaly is a rare and slowly progressive disease characterized by the abnormal growth of bones and enlarged hands, feet and facial features. Acromegaly also affects internal organs and can typically include symptoms such as headaches, joint pain and fatigue.
Acromegaly is often caused by a tumor in the pituitary gland, which leads to the excess production of growth hormone and insulin growth factor-1. When uncontrolled, acromegaly worsens patients’ quality of life or can heighten their risk of death.
Octreotide, the active ingredient of CAM2029, is a peptide drug that is well-known as an acromegaly therapy. It works by mimicking the function of somatostatin, a naturally occurring hormone, and activating its corresponding receptor. This mechanism of action helps suppress growth hormone, addressing the underlying cause of acromegaly. Octreotide also lowers the levels and activity of insulin and glucagon.
The pharma industry has long taken advantage of octreotide’s therapeutic effects for acromegaly. Sun Pharmaceuticals won FDA approval for Bynfezia Pen (octreotide acetate) in 1988, which is indicated for the three-times-daily subcutaneous treatment of acromegaly. The drug is also approved for carcinoid tumors and vasoactive intestinal peptide tumors.
Camurus’ investigational formulation is an extended-release version of octreotide that would allow for once-monthly dosing, giving patients a more convenient treatment option. In the Phase III ACROINNOVA 2 study, CAM2029 led to better biochemical control of acromegaly versus the current standard of care. Camurus’ injection also improved quality of life and symptom burden.
