Rare Diseases

Our strategy

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. Developing new treatments sooner, better, faster for them is our goal. Yet, relatively common symptoms can hide underlying rare diseases leading to misdiagnosis and delayed treatment. We are focused on challenging this status quo with our dedicated focus on the discovery of new drugs and continued education about the signs and symptoms to aid early diagnosis.

We understand that competing in the rare diseases space requires a distinct operating model and specific capabilities such as:

Deep knowledge of the patient ecosystem
Close collaboration with patient advocacy groups
Efficient patient enrolment for clinical trials
Extensive scientific expertise to interact with physicians who treat rare disease patients
Ability to provide tailored patient services and education programs

We are proud to have developed these attributes at Ipsen and strongly believe that we will continue to add medical value for rare disease populations in the years to come.

Strategy

We have a social contract with society; that means society is counting on us to accelerate the access of drugs to meet patients in need.

David Meek, CEO

Key Facts

In 2018, sales of our rare diseases drugs totaled €70m, equivalent to 3.9% of our total sales.

A disease or disorder is defined as rare in Europe when it affects fewer than one in 2,000 people.

In the US, the definition applies to conditions that affect fewer than 200,000 people in the country at any given time.

An estimated 350+ million patients are suffering from one of over 7,000 rare diseases globally.

50% of the people affected by rare diseases are children.

A total of 30% of rare disease patients die before the age of 5 and rare diseases are responsible for 35% of deaths in the first year of life.

Some 95% of rare diseases do not have a single FDA-approved drug treatment and as a result represents an area of high unmet medical need.

SOMATULINE®

SOMATULINE® is 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. Some 69,000 patients worldwide are affected by this disease. We are currently developing extended-release formulations of SOMATULINE® so that fewer injections would be required.

NUTROPIN AQ®

INCRELEX®

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. It is the only drug available at the global level for this indication. In 2017, a new Ipsen manufacturing site was approved by both the EMA and FDA to produce INCRELEX®.

Other Drugs

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. There is a potential opportunity for greater use of this treatment in the European Union, China and Russia.

Our rare diseases pipeline

Further developing our presence in rare diseases constitutes a natural path forward for Ipsen. In the future, we will continue to expand in this area with high unmet need, leveraging expertise from development to commercialization to establish leadership positions and provide innovative treatments.

We will strengthen our pipeline and portfolio through targeted business development efforts, considering multiple factors in our assessment such as disease prevalence and severity, availability of treatments and the commercial and technological fit with Ipsen.

In particular, we will strengthen our portfolio with a particular emphasis on treatments for children. We believe we need to provide our caregivers and our physicians with the ability to treat these patients with new options and the launch of new trials is crucial this. This is why we believe the challenges are diminishing but the opportunities are growing.

Partnerships

By partnering with Ipsen, you will gain access to a like-minded global biopharmaceutical company with powerhouse development and commercialization teams. We are willing to take bold steps to translate your science and innovations into impactful products for patients in need.

Learn more

Partnerships with patients and healthcare providers

Ipsen’s support for acromegaly patients 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®. The website includes advice on issues relating to 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.

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