Our Commitment to Rare Disease
Working with the rare disease community, we strive to better understand the challenges faced by those living with rare diseases and their families, and then use our expertise to help address them. We are inspired by the strong and resilient rare disease communities around the world, driving us to work even harder to develop innovative treatments to improve their lives. We actively listen to and learn from their experiences and integrate their insights into our efforts throughout the development process.
We have been focused on developing potential treatments for rare diseases for many years, and it remains a core part of our strategy. We have invested significantly in research and development and leveraged our scientific expertise to bring scientific advancements to patients. By conducting first-in-the-world clinical trials, we help continue the search for life-changing medicines.
Targeted research in bone disease, endocrinology, and other areas with high unmet medical needs.
Plans to expand our work into additional disease areas with unmet need, established and emerging modalities and gene-based therapies, and all stages from late-preclinical to marketed products.
Our Areas of Expertise
Our Rare Disease Pipeline
At Ipsen, our research decisions are guided by patient outcomes. Emphasizing a culture of open innovation, we focus our R&D efforts on accelerating prioritized internal projects while actively sourcing external assets in clearly defined segments. As we target differentiated medicines in our core areas, including rare disease, we will support innovative and differentiated therapeutics, bolstering our external sourcing model while expanding our portfolio. We will also maximize the value of our current pipeline assets.
As a global biopharmaceutical company with a focus on innovation and specialty care, we have the ambition to build a robust, valuable, and sustainable portfolio of medicines to meet those living with rare diseases and their healthcare teams’ needs. We have a focused approach and prioritize best-in-class collaborations for new, emerging products in rare disease – an area where we have proven experience and global capabilities along with a strong network of partners.Learn more about our pipeline
Working with Patients, For Patients
Ipsen’s priority is to improve the lives of patients, their caregivers, their communities and, ultimately, society. By putting people living with health conditions at the center of everything we do, we develop support solutions that meet the real needs of those living with rare diseases and can help make a meaningful difference and improve their lives. We have built strong, long-term partnerships with patient organizations and the rare disease community, and we frequently invest in efforts to increase disease awareness and education, including:
Rare Disease Patient Organizations
We collaborate with rare disease organizations around the world to ensure we are understanding their perspectives and responding to their needs. We also collaborate to raise awareness and increase understanding of rare and ultra-rare diseases.
The World’s Rarest Dictionary Project
The World’s Rarest Dictionary is a project that aims to encourage others to listen, understand and reflect on the experiences of people affected by rare diseases. From those who actually live with a rare disease, to their family and friends, caregivers and researchers working across these areas.
Focus on FOP
Focus on FOP is a website for people living with FOP or their families and caregivers. It features patient experiences , videos, and images, as well as more information about the condition. There are also links to national patient organization websites for more information and support.
The Acromunity website was co-developed with patients and healthcare professionals to provide resources and support for those living with acromegaly. The website also provides information about patients support groups available in individual countries.
Living with NETs
The Living with NETs website provides resources and support for those living with neuroendocrine tumors.
We believe that great partnerships create great possibilities. We are on a mission to deliver at least one new molecular entity or meaningful indication each year and collaborations with other like-minded innovators are key for the next phase of our growth.
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