Rare Diseases

In the field of rare diseases, Ipsen takes a patient-focused approach to improving the lives of people affected by rare disorders and develops high-quality, innovative treatments that address unmet needs. Our goal is to support patients with drugs, services and solutions across the entire continuum of care, from diagnosis to treatment follow-up.



Ipsen has been active in the rare diseases field for many years and intends to bring new solutions to patients, notably children, in the future.

Somatuline® is also used for the long-term treatment of acromegaly, a rare disease caused by excess growth hormone production as a result of a tumor in the pituitary gland, in patients who cannot be treated with surgery or radiation. We are currently developing extended-release formulations of Somatuline® so that fewer injections would be required.

NutropinAq® is a liquid formulation of recombinant human growth hormone administered with the NutropinAq® Pen.
Available in more than 20 countries, notably in Europe and Australia, it is indicated for the treatment of growth failure stemming from various origins.

Increlex® is a recombinant insulin-like growth factor (IGF-1) that treats growth delay in children. If IGF-1 is not present in sufficient quantities, the patient will not reach normal stature, despite having normal or high growth-hormone levels. As a result, these children do not respond adequately to growth hormone treatment.
Increlex® has obtained orphan drug status based on the low incidence of the disease, which affects fewer than five people per 10,000.

Ipsen also offers a number of other important solutions for patients with other debilitating or life-threatening conditions. For instance, Decapeptyl® (triptorelin) is also approved for the treatment of central precocious puberty (CPP) endometriosis, uterine fibroma, and in vitro fertilization.


Ipsen’s support for acromegaly patients also goes beyond treatments. The Acromunity.com website, launched in 2017 in conjunction with patients and healthcare professionals, offers a platform, that delivers content, tools and services to match their needs, from the time of first symptoms to years after diagnosis.

In the United States, Ipsen supports IPSEN CARES™ (Coverage, Access, Reimbursement and Education Support), a program that assists patients in overcoming obstacles to start or continue treatment with Somatuline® for gastroenteropancreatic NETs and acromegaly, as well as Increlex® and Dysport®, including coverage access, distribution and financial concerns.

In Europe, the Group has set up INKEP (Ipsen Network of Knowledge Exchange Program) for small groups of physicians specializing in pediatric endocrinology. It combines scientific presentations, case discussions and interactive sit-in clinic visits.

Other initiatives in pediatric endocrinology include APPRI in France, a personalized training program for patients that helps increase their autonomy at home during treatment with the recombinant growth hormone NutropinAq® and the NutropinAq® injection pen, which improves treatment compliance.

Furthermore, Ipsen is committed to promoting transparent and evidence-based discussions between stakeholders on policies concerning the management of conditions on which it focuses – including NETs, rare diseases and urological cancers.

€75.1 M

Sales in endocrinology in 2017

(3,9% of sales in 2017)