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At its April 2025 meeting, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) gave conditional marketing authorization for Oczyesa (octreotide) for the maintenance treatment of adults with acromegaly who have responded to and tolerated treatment with somatostatin analogs.

Conditional marketing authorization is granted to a medicinal product that fulfills an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.

Acromegaly is characterized by excessive growth hormone (GH) and insulin-like growth factor 1 (IGF-1) and affects quality of life and mortality. 

Oczyesa was submitted in a hybrid application, which relies in part on the results of preclinical tests and clinical trials of an already authorized reference product and in part on new data. In making its recommendation, the CHMP highlighted that the benefits of Oczyesa were the reduction and, in many cases, normalization of IGF-1 and GH levels in patients with acromegaly, as shown in two phase 3 studies.

Improved Symptom Control, Quality of Life, and Satisfaction

The first study was a 24-week, randomized, double-blind, placebo-controlled, multicenter study, ACROINNOVA 1, and the second one was a 52-week, open-label, multicenter study, ACROINNOVA 2.

ACROINNOVA 1 set out to assess the superiority of Oczyesa vs placebo for biochemical control in patients with controlled acromegaly. Patients were randomly assigned (2:1) to once-monthly Oczyesa or placebo. At week 22/24, patients treated with Oczyesa demonstrated superior response rates vs placebo for IGF-1 (72.2% vs 37.5%) and combined IGF-1/GH (70.0% vs 37.5%). The researchers said that Oczyesa provided a “convenient and effective treatment option for acromegaly, with superior biochemical control vs placebo.” They added that symptom control, quality of life, and satisfaction were improved from the baseline standard of care.

The persistence of mean IGF-1 values and reduction of symptoms was confirmed over 52 weeks in the ACROINNOVA 2 study. Furthermore, this study showed improvements in symptoms, quality of life, and treatment satisfaction scores after 52 weeks of treatment with Oczyesa compared with the standard of care at the study baseline.

Convenient Self-Administration

The active substance of Oczyesa is octreotide hydrochloride, a synthetic derivative of naturally occurring somatostatin, a hypothalamic hormone. It works by reducing and normalizing IGF-1 and growth hormone levels in patients with acromegaly. It achieves this by inhibiting the overproduction of growth hormone.

Oczyesa is a long-acting subcutaneous depot formulation — a 20 mg prolonged-release solution for injection in a prefilled pen — delivered once monthly and designed for self-administration.

The most common side effects of Oczyesa include gastrointestinal disorders, nervous system disorders, hepatobiliary disorders, metabolism and nutritional disorders, and injection site reactions.

Since Oczyesa was designated as an orphan medicine during its development for use against a rare, life-threatening, or chronically debilitating condition, or for economic reasons that would have been unlikely to have been developed without incentives, the EMA said that it would now review the information available to date to determine if the orphan designation can be maintained.

Rob Hicks is a retired NHS doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online. He is based in the UK.