Ipsen in Rare Diseases
At Ipsen, we are driven to develop and enable timely access to innovative therapies and provide solutions for people living with debilitating and / or life threatening rare conditions.
Working together with the rare diseases community, we strive to better understand the challenges faced by patients and their families and then build on our expertise to address them.
We want people living with rare diseases to know we support them throughout their entire journey for better management of their conditions. Our ultimate goal is to leave no patient behind.
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.
Our commitment to Rare Diseases
We have been active in the rare diseases field for many years, bringing scientific advancements to patients, investing significantly in our agile R&D model and leveraging our global expertise and local presence. Our people are committed to making innovative new treatments available to people with rare diseases as quickly as possible.
We know that embracing co-creation is the most effective way to address the challenges of living with rare diseases. This is why we listen to patients, their families, carers and healthcare providers, integrating their insights in to our efforts from the early stages of R&D.
It is through trust, collaboration and strategic partnerships that we will achieve the best results for rare disease patients around the world.
Our areas of expertise
Our Rare Diseases pipeline
At Ipsen, we are focused on delivering on our growth strategy. Core to the strategy is attracting the most promising external innovation so that we can deliver impactful new therapies for people affected by cancer, neurological and rare diseases.
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 of high unmet medical needs, leveraging expertise from development to commercialization to establish leadership positions and provide innovative treatments.
In particular, we will strengthen our portfolio with a particular emphasis on treatments for children. We believe we need to provide our caregivers and physicians with the ability to treat these patients with new options and the launch of new trials is crucial this.
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.
Clementia, an Ipsen Company
In April 2019, Ipsen acquired Clementia, a Canadian clinical stage biotech company focused on innovative new treatments for people with rare bone disorders and other diseases.
By bringing two passionate and patient-centric teams together, it significantly enhances our ability to treat rare diseases in children and adults, specifically fibrodysplasia ossificans progressiva (FOP) – an ultra-rare, severely disabling disorder characterized by bone that forms outside the normal skeleton, in muscles, tendons or soft tissue as well as multiple osteochondromas (MO) – a rare, severely disabling, progressive, chronic disease in which multiple benign bone tumors develop on bones.
Palovarotene – our late-stage clinical asset – is an activator of the retinoic acid receptor gamma (RARy) selective agonist, which is required for muscle repair and skeletal growth. In patients with FOP, the activation of this receptor decreases the communications between proteins that promote abnormal bone formation.
This treatment option has received orphan drug designation for FOP and MO from the FDA, fast track and breakthrough therapy designations for FOP from the FDA and orphan status for FOP from the European Medicines Agency (EMA).
We are dedicated to advancing palovarotene as quickly as possible for the benefit of the greatest number of patients globally, who have been waiting for a treatment to alleviate their suffering.
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.