Patrick Lewis, PharmD, Senior Director, Medical Affairs at Amryt Pharma discusses the latest data presented at ENDO 2023 on oral octreotide to treat acromegaly.



Acromegaly is a rare endocrine disorder due to excess levels of growth hormone (GH). A benign tumor on the pituitary gland causes the excess of GH in many cases.

Common symptoms include:

  • subtle skeletal overgrowth
  • soft tissue swelling (tongue, heart, kidney, colon, and vocal cords)
  • unusually prominent forehead and heavy brow ridge
  • protruding lower jaw
  • overbite
  • skin thickening
  • increased shoe and ring size
  • hypertension
  • increased perspiration
  • headaches
  • paresthesia
  • sexual dysfunction

However, due to the insidious nature at which acromegaly symptoms develop, there is an average diagnostic delay of 5.5 years.   

There are currently four orphan drugs approved to treat acromegaly. They either stimulate somatostatin receptors (octreotide, octreotide-oral, pasireotide) or inhibit GH receptors (pegvisomant). 

New Data

At ENDO 2023, new data was presented that can help predict treatment response to oral octreotide. The abstract, by Maria Fleseriu et al, was a posthoc analysis of the baseline characteristics of patients enrolled in the pivotal MPOWERED trial (Lancet Diabetes Endocrinol. 2022;10:102-111) to determine if any characteristics could predict treatment response. Patients in the study were randomized to receive oral octreotide or injectable somatostatin receptor ligands ( octreotide or lanreotide). Of all the baseline characteristics examined (e.g., baseline IGF-I levels, GH levels, duration of acromegaly, history of surgery and/or radiation, baseline pituitary adenoma size, and residual postoperative pituitary adenoma size), only baseline IGF-I level when being treated with injectable somatostatin receptor ligands was a predictor of subsequent oral octreotide response. 

In a second abstract, Eliza Brevoort Geer et al compared patients’ and physicians’ questionnaire results and noted that in In the biochemically controlled group, 73% (107/147) of patients and 72% (106/147) of physicians reported symptoms as well-controlled. However, there was a large discrepancy between the two groups regarding individualized symptoms. Memory problems, fatigue, and joint pain were the most frequent symptoms reported at baseline by biochemically controlled patients that were not reported by their matched physician (33, 25, and 18% of patients, respectively). Further, in the 45 biochemically controlled patients that rated any of their symptoms as severe, only 5 matched physicians reported the same rating of severity. This study illustrates the need for better communication between clinicians and patients. 


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