Camurus (NASDAQ STO: CAMX) announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending market authorization for Oczyesa®, a subcutaneous octreotide depot (CAM2029), for the maintenance treatment of adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues.
The European Commission is expected to make a final decision on marketing authorization by mid-2025, potentially offering a new treatment option for patients with this rare endocrine disorder.
"Oczyesa has the potential to advance the standard of care for patients living with acromegaly by enhancing octreotide plasma exposure and enabling easy and convenient once-monthly self-administration by patients using an autoinjector pen," said Fredrik Tiberg, President & CEO at Camurus.
Clinical Evidence Supporting Approval
The CHMP's positive opinion is based on data from a comprehensive clinical program comprising seven studies, including two pivotal Phase 3 trials. The ACROINNOVA 1 study demonstrated that Oczyesa treatment resulted in a significantly higher proportion of patients achieving normalized insulin growth factor-1 (IGF-1) levels compared to placebo.
In the ACROINNOVA 2 study, researchers confirmed the persistence of normalized mean IGF-1 values and symptom reduction over a 52-week period. This study also showed improvements in symptoms, quality of life, and treatment satisfaction scores after 52 weeks of Oczyesa treatment compared to standard of care at baseline.
The most commonly reported adverse events included gastrointestinal disorders, nervous system disorders, hepatobiliary disorders, metabolism and nutritional disorders, and injection site reactions.
Addressing Unmet Needs in Acromegaly
Acromegaly is a rare, progressive disease affecting approximately 60 people per million. It is typically caused by a pituitary gland tumor that produces excess growth hormone, stimulating increased insulin growth factor-1 (IGF-1) levels. This hormonal imbalance results in abnormal growth of bone and tissue, enlarged hands, feet, and facial features, as well as symptoms including fatigue, joint pain, headache, visual field defects, excessive sweating, and paresthesia.
Inadequate biochemical and symptom control can significantly impact patients' quality of life and mortality rates. Current treatments often require intramuscular injections administered by healthcare professionals, creating barriers to optimal disease management.
Innovative Drug Delivery Technology
Oczyesa represents a significant advancement in acromegaly treatment, demonstrating approximately five-fold higher bioavailability compared to currently approved long-acting intramuscular octreotide formulations.
The product is formulated using Camurus' proprietary FluidCrystal® technology, designed for convenient once-monthly subcutaneous self-administration using a pen with a hidden, small-gauge needle. Unlike some existing treatments, Oczyesa can be stored at room temperature without refrigeration, potentially improving treatment adherence and convenience.
"This new formulation addresses several key challenges in acromegaly management by improving drug bioavailability and enabling patient self-administration," explained Dr. Diego Ferone, lead investigator in the ACROINNOVA clinical program. "The data show that these improvements translate to better biochemical control and quality of life outcomes for patients."
Broader Development Program
Beyond acromegaly, CAM2029 is under development for two additional indications: gastroenteropancreatic neuroendocrine tumors (GEP-NET) and polycystic liver disease (PLD). The drug has received orphan drug designation for acromegaly in the EU and for polycystic liver disease in both the EU and US.
Camurus continues to expand its portfolio of long-acting medicines based on its FluidCrystal® technology platform, with products targeting dependence, pain, cancer, and endocrine diseases.
The company, headquartered in Lund, Sweden, maintains operations across Europe, the US, and Australia, with shares listed on Nasdaq Stockholm under the ticker CAMX.
Market Impact and Future Outlook
If approved by the European Commission, Oczyesa would enter a market where current treatment options include first-generation somatostatin receptor ligands such as octreotide and lanreotide, which typically require intramuscular administration by healthcare professionals.
The approval would represent a significant milestone for Camurus as it continues to expand its portfolio of innovative drug delivery systems for chronic conditions. The company's approach focuses on improving treatment outcomes through enhanced bioavailability and patient-friendly administration methods.
Industry analysts suggest that improvements in treatment convenience and efficacy could significantly impact patient adherence and disease management in this rare disease population, potentially establishing a new standard of care for acromegaly maintenance therapy.
