Endocrinology is one of Ipsen's key Specialty Care pillars, focusing on endocrine oncology (neuroendocrine tumors or NET), pituitary pathologies (acromegaly), and growth disorders (growth hormone and IGF-1 deficiencies).

Innovative medicines

Ipsen continues to develop innovative medicines to address the unmet medical needs of patients. 2014 was an important year with several major clinical and regulatory advances for the treatment of patients with gastroenteropancreatic neuroendocrine tumors. In addition, our product to treat patients with severe primary IGF-1 deficiency was resupplied in the United States and in Europe.

Ipsen also took part in many initiatives to support clinicians and patients. Late diagnosis of acromegaly and NETs, the consequences for patients and the efficacy of their treatment is a public health concern.

Somatuline®, one solution for two rare conditions

Somatuline® is a formulation for injection that inhibits several endocrine, exocrine and paracrine functions, involved in two rare diseases: acromegaly and neuroendocrine tumors. Somatuline® has also shown an antitumoral activity that is believed to be mediated via both direct and indirect mechanisms. Direct mechanisms involve the activation of somatostatin receptors on tumor cells leading to modulation of intracellular signaling transduction pathways, whereas indirect mechanisms include inhibition of mitogenic growth factors such as insulin-like growth factor (IGF), as well as inhibition of tumor angiogenesis through interactions with somatostatin receptors on endothelial cells and monocytes. Somatuline® is marketed in over 55 countries (25 in Europe) for the treatment of acromegaly and neuroendocrine tumors.


Acromegaly, which is often mistaken for gigantism, is a hormonal disorder, marked by a thickening and widening of the hands and feet as well as an alteration of facial features, which set in slowly over time.  These symptoms are related to a pituitary tumor which produces excess growth hormone (GH). Acromegaly is rare: 40-70 cases per million).

Neuroendocrine tumors

Neuroendocrine tumors are rare diseases (±1% of digestive tumors). These tumors, most commonly found in the gastrointestinal tract, secrete abnormally high quantities of hormones which can cause diarrhea and flushing. As a result of the absence of specific symptoms, the disease is often diagnosed at a late stage.

Nutropin Aq®, a treatment for growth hormone deficiency

Nutropin Aq® is a liquid formulation of recombinant human growth hormone administered using the NutropinAq® Pen. Growth hormone is involved in physiological processes such as growth in stature and bone development in children. Nutropin Aq® was approved in more than 30 countries as of the end of 2014.

Nutropin Aq® is indicated for the following:

  • long-term treatment of growth failure in children due to inadequate endogenous growth hormone secretion;
  • long-term treatment of growth failure associated with Turner syndrome;
  • treatment of growth failure in prepubescent children associated with chronic renal failure prior to kidney transplantation;
  • treatment of adults with either childhood or adult onset growth hormone deficiency.

Increlex®, an orphan drug

The active substance in Increlex® is a recombinant insulin-like growth factor of human origin (IGF-1). IGF-1 is the direct hormonal mediator of growth, and must be present for normal growth of bones and cartilage in children. In severe primary IGF-1 deficiency, serum IGF-1 levels are low in spite of normal or high growth hormone levels. If IGF-1 is not present in sufficient quantities, the child will not reach normal stature. These low IGF-1 levels are usually due to growth hormone resistance associated with mutations affecting the GH receptors and the post-GH receptor signaling pathways, or defects in IGF-1 gene expression. This explains why these children cannot respond adequately to exogenous growth hormone treatment.  As this is a rare condition, Increlex® was granted the status of orphan drug.

Numerous partnerships

The Group partners with medical societies (including ENETS, ENDO and ASCO), patient groups (such as the International NET Cancer Association), as well as scientific meetings and conferences. Ipsen is also involved in creating networks of experts to promote international dialog between specialists. In many countries, the Group cooperates closely with neuroendocrine tumor patient groups to raise awareness of these disabling conditions and make progress towards ealier diagnosis. Ipsen also suppports the International Neuroendocrine Cancer Alliance (INCA) for World NET Cancer Day held on November 10, every year.

In the United States, Ipsen introduced IPSEN CARESTM (Coverage, Access, Reimbursement and Education Support), a program to support access to Somatuline® Depot for treatment of GEP-NETs and acromegaly, Increlex® and Dysport®. IPSEN CARESTM assists in overcoming obstacles to start or continue treatment of the products including coverage access, distribution and financial concerns. In Australia, Ipsen launched its ASSIST scheme to train patients and caregivers to facilitate administration of Somatuline®, offering the flexibility of self-injection at home or injection by a relative. Other initiatives for acromegaly patients in the United Kingdom and in France were carried out, such as the launch of a website developed in cooperation with the French Endocrinology Society.

Ipsen supported the launch of a national Acromegaly Day in Poland on May 12, 2014. Our engagement in this area aims to increase awareness and knowledge about acromegaly among medical professionals and the general public.

Updated on 31 July 2015