Our Commitment to Rare Disease
Ninety-four percent of rare medical conditions still have no treatment. Today our Rare Disease teams are more committed than ever to changing that. We leverage scientific expertise, our global presence, and a robust external innovation strategy, plus first-in-the-world clinical trials to develop life-changing rare disease therapeutics.
Listening to people living with rare diseases
With unwavering focus on patient care, we strive to understand the challenges faced by those living with rare diseases. We learn from their experiences and integrate their insights into our efforts throughout the development process. As a result, we are gaining momentum toward finding treatments for rare diseases including musculoskeletal (bone), metabolic, endocrine, gastrointestinal (liver) and neuromuscular disorders.
Learn moreChallenging treatment paradigms
In 2023, Ipsen will be reporting on a Phase III clinical trial with critical implications for treating an autoimmune liver disease, Primary Biliary Cholangitis (PBC), and its debilitating symptoms.
Unique challenges require unique solutions
In 2023, we further strengthened our Rare Disease portfolio with promising treatments and investigational assets for pediatric and adult rare liver diseases.
An unwavering fight to meet patient needs
94% of rare diseases still lack approved treatments.
Rare but not alone
350-400 million people are impacted by one of 7,000 rare diseases globally.
Innovating for patients with rare diseases
The rare disease clinical pipeline has more than doubled since 2020.
Continuous pioneering in rare disease
A core part of our strategy, Ipsen has focused on developing transformational treatments for rare diseases for many years.
Bone disorders
FOP causes permanent and continuous abnormal new bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as heterotopic ossification (HO). It is an ultra-rare genetic disorder, characterized by inflammatory flare-ups that begin in childhood HO is irreversible and leads to severe functional limitations in mobility, progressive and cumulative disability, and shortened life expectancy. On average, patients are diagnosed at the age of 5, and their median life expectancy is 56. Around 900 people worldwide are currently living with an FOP diagnosis. Ipsen is harnessing novel mechanisms of action through the development of two investigational assets to bring potentially transformative treatments to those living with FOP.
Growth disorders
Acromegaly is a rare disorder caused by a benign (noncancerous) tumor of the pituitary gland, called an adenoma, that promotes too much growth hormone, or GH, to be released into the bloodstream. As a consequence, the body makes more of another hormone, insulin-like growth factor 1 (IGF-1).
Normally, IGF-1 works with GH to help control growth of many tissues in the body. With too much IGF-1 and GH, the body can undergo abnormal growth of bones, cartilage and other body tissues. While the more outward signs are enlarged hands, feet and face, more serious problems may also develop.