Ipsen’s financial results for the first half of 2025 will be published on 31 July 2025 at 07:00 CET.
An investor and analyst conference call will follow at 13:00 CET.
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.
Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries, and our partnerships around the world, enable us to bring medicines to patients in more than 100 countries.
Discover Ipsen in 3 pages; our medicines and key figures, our objectives and our strategy.
If you are experiencing difficulties registering for the investor and analyst conference call, please contact: khalid.deojee@ipsen.com
At just nine years old, Suzanne is a bright, creative young girl who loves dancing, drawing, exploring the woods, and watching birds. Despite living with Alagille syndrome (ALGS), a rare genetic condition, she embraces life with enthusiasm and curiosity.
ALGS primarily affects the liver but can also impact other multiple organs in the body, including the heart, brain, bones and eyes1. In babies born with ALGS, the liver has fewer bile ducts, which means bile and toxins can’t flow out as normal. Instead, they build up in the body – a condition known as cholestasis2. This buildup is responsible for one of the most common and debilitating symptoms of ALGS – pruritus, or intense itching.
“This illness gets on my nerves, and I just want it to go away. To not be a part of my body.” – Suzanne
Shortly after her birth, Suzanne’s mother, Céline, sensed something wasn’t right. Despite a healthy pregnancy and delivery, Suzanne cried often and had a yellow complexion. A chance comment from a speech therapist confirmed Céline’s concerns, leading to a diagnosis of ALGS.
One of Suzanne’s greatest challenges has been severe pruritus, a relentless itch, that began when she was just nine months old. The discomfort was so intense that Céline often had to wake several times each night to comfort her, resulting in years of exhaustion and emotional strain for the whole family. Like many caregivers of children with rare diseases, Céline navigates the daily challenges of balancing care with the rhythms of family life.
“I had to get up almost every night, even several times in a night, to calm her down and soothe her. And that was very difficult. Because you have to be able to get through the day too, and then getting up at night inevitably makes you tired, even as a caregiving mother” – Céline
Today, with effective care, Suzanne continues to find joy in everyday activities. Patient organizations like AMFE (Association Maladies du Foie depuis l’Enfance) have also been a lifeline for the whole family, offering vital support, connection, and community for those living with rare liver diseases.
At Ipsen, we work closely with the rare liver disease community to understand what matters most. By partnering with the community, we strive to raise awareness, shorten the path to diagnosis, and reduce the emotional and physical burden families like Céline and Suzanne’s carry. For us, making a difference in their lives means everything.
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The Ipsen S.A. 2025 Annual General Meeting took place on 21 May 2025, in Paris, France.
21 May 2025 – 1 mins read
Annual General Meeting of Ipsen S.A. held on 21 May 2025
On 21 May the Annual General Meeting has been filmed and transmitted live. Click on the link below to watch the replay.
Watch the replay
The Q1 2025 sales update have been published on April 16, 2025 at 7H00 and a conference call took place at 14H00 CET.
16 April 2025 – 1 mins read
Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay.Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma.Confirmation of full-year 2025 financial guidance. PARIS,…
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY).
Our vision is to be a leading global mid-sized biopharmaceutical company with a focus on transformative medicines in Oncology, Rare Disease and Neuroscience. Discover Ipsen in two pages; our products and key figures, our objectives and our strategy.
If you are experiencing difficulties registering for the investor and analyst conference call, please contact: khalid.deojee@ipsen.com
Professor David Jones and Professor Valerie McLin were compensated by Ipsen for their time.
Rare cholestatic liver diseases are conditions that can impact both children and adults. Three such conditions—Primary Biliary Cholangitis (PBC), Progressive Familial Intrahepatic Cholestasis (PFIC), and Alagille Syndrome (ALGS)—share commonalities yet are unique in their causes, manifestations, and challenges for patients and their families.1, 2 Understanding these diseases is crucial for better diagnosis, treatment, and overall care.3
PBC is a rare, autoimmune, cholestatic liver disease with increasing prevalence worldwide. In people with PBC, the body attacks and gradually destroys the liver’s small bile ducts.3 If left untreated, bile and toxins may build-up (cholestasis), leading to scarring of the liver (cirrhosis) and eventual liver failure.4,5,6
Accumulation of bile may cause liver damage, symptoms like fatigue and intense itching. PBC is often underdiagnosed due to its subtle symptoms, making early recognition and intervention essential for managing the disease.7
PFIC on the other hand, refers to a set of rare genetic conditions where the liver has difficulties releasing bile acid needed to digest fats and oils. The buildup of bile can cause great harm to the liver, potentially leading to liver failure.8 Although PFIC can present itself later in life, it generally manifests and is most aggressive in infants and young children.9, 10
ALGS is an inherited condition that can affect the liver, heart, brain, blood vessels and bones.11, 12, 13 Children with these conditions often require nutritional support and face frequent medical visits, impacting their schooling and social development.14
Despite their differences, these diseases share important commonalities. A key symptom across all of them is pruritus (itching), which can be overwhelming, especially in children. 15, 16, 17 For people living with these diseases, the itch is far more than a mild irritation; it can be all-consuming, disrupting daily life and causing significant distress. In addition, fatigue is also a symptom that causes great burden particularly for people living these conditions, with many experiencing debilitating tiredness that affects concentration and social interactions.
Ipsen is committed to raising awareness of rare cholestatic liver diseases and bringing treatment options to people living with these conditions and their families. Advances in medical research are leading to more treatments and more informed symptom management. 18 However, awareness of these conditions remains low among patient communities and healthcare professionals (HCPs) throughout the world. Increased discussions about symptoms are important to help people living with rare cholestatic liver diseases understand and manage their symptoms and achieve timely diagnosis.
As we look to the future, a collaborative, patient-centered approach is key to managing these complex diseases.
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An article by Jennifer Schranz, Senior Vice President and Global Head of Rare Diseases at Ipsen
In storytelling, you might hear of a superhero who squeezes coal with immense strength and heat, instantly creating a flawless diamond. While fiction, it’s true that diamonds are rare and formed under precise conditions over millions of years. For me, this analogy resonates deeply with the development of rare disease medicines—an arduous, highly complex, and time-intensive process that transforms scientific discovery into potentially life-changing treatments.
At the Orphan Drug and Rare Disease (ODRD) Congress, I addressed the myths and challenges surrounding rare disease innovation. More importantly, I spoke about how we, as an industry, must collaborate to ensure people living with rare diseases have access to the treatments they urgently need. Rare disease drug development is uniquely complex—marked by small patient populations, unclear clinical endpoints, and slow recruitment. Yet, there are other barriers that lie beyond the laboratory: regulatory frameworks, pricing, and reimbursement policies that do not fully account for the distinct challenges of rare disease R&D.
Of the more than 10,000 known rare diseases, 95% still lack an approved treatment.1 For patients and families, every breakthrough represents more than just a therapy—it offers hope, better quality of life, and more time with loved ones. Yet, financial and structural barriers can slow innovation and limit access to treatments. Without a proactive approach, progress will remain out of reach for too many.
Developing a rare disease medicine is like mining for diamonds—nature may create something extraordinary, but extracting and refining it requires immense effort. Just as only a small percentage of mined diamonds are suitable for fine jewelry, many promising drug candidates never reach those who need them. This is why we must rethink how we develop and evaluate treatments for rare diseases.
To bring more treatments to people with rare diseases, we need a shift in how we approach drug development. A case-by-case model for each disease is unsustainable. Instead, we must embrace innovation across three key areas:
Across all of these, collaboration is key. By building strong partnerships between industry, academia, patient organizations, and regulators, we can develop a shared vision for rare disease innovation. The International Rare Diseases Research Consortium (IRDiRC) aims to support the development of 1,000 new rare disease therapies by 2027.2 To get there, we need sustained investment, incentives, and fresh thinking about development and access.
Incentives for orphan drug development have long supported rare disease innovation, fostering investment and research in this critical area. In recent years, many new treatments have emerged for rare diseases, yet shifting policies and evolving economic considerations could slow this momentum.
A thriving research ecosystem doesn’t happen by chance—it requires careful nurturing. If incentives decline, we may see fewer translational research programs, slower clinical trials, and weakened industrial partnerships. This isn’t just about the pharmaceutical industry; it’s about ensuring people with rare diseases continue to have hope for new treatment options.
What drives me most is making a real difference in the lives of people living with rare diseases—not just by developing treatments, but by ensuring they receive the support and access they deserve. The impact of rare diseases is profound, and we cannot afford to slow progress.
So where do we go from here? Let’s create an environment that accelerates breakthroughs and prioritizes access. Like diamonds, every new therapy results from pressure, persistence, and the right conditions—let’s ensure these rare and valuable treatments reach the people who need them most.
In the latest installment of our ‘A Life in Science’ series, we’re excited to introduce Crystel Ogier, our Clinical Biomarker Director, who leads the awareness and implementation of companion diagnostics at Ipsen. Crystel’s work is a prime example of how cutting-edge science is making a real impact in shaping the future of drug development and improving patient outcomes.
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Next in our ‘A Life in Science’ series, Dorinne Desposito, Predictive Toxicology Project Manager, shares her insights on driving innovation through creative thinking and the power of shared learning within our dedicated teams. Dorinne’s work exemplifies how cross-functional collaboration at Ipsen is advancing science, making a meaningful impact, and ensuring that science remains both fun and motivating.
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An article by Philippe Ghyssels, Vice President Corporate and Global Public Affairs
For individuals living with a rare disease, every day without treatment is a day too long. At the World Evidence, Pricing and Access (WEPA) congress in Amsterdam, I participated in a panel discussion on long-term access to rare disease innovation. The conversation reminded me that behind every policy, negotiation, and delay are real people whose lives depend on timely access to treatment.
Innovation in rare diseases has come a long way, but as we continue to make progress, we need to ensure that access keeps pace with innovation. A new treatment can only change lives if it consistently reaches the people who need it, when they need it.
And yet, timely access isn’t always straightforward—rare disease treatments don’t fit neatly into the traditional pharmaceutical development and access model. The orphan drug business model is unique—it requires a thoughtful balance between fostering innovation and making therapies available equitably and without delay. While many advances have been made, there are still areas where we can work together to improve:
Rather than seeing these as barriers, we should view them as opportunities to refine and optimize access pathways—so that innovation translates into impact, faster and more consistently.
How can we move forward? Ensuring that rare disease treatments reach people in a sustainable, long-term way requires collaboration and fresh thinking. For me, key areas of focus include:
One of my biggest takeaways from the World EPA Congress is that there is a strong, shared commitment to improving rare disease access. Policymakers, healthcare professionals, regulators, patient groups, and industry are all working toward the same goal—making sure that people with rare diseases can benefit from innovation in a sustainable and timely way.
Rather than focusing on what isn’t working, we should channel our efforts into what we can build together. By aligning incentives, fostering collaboration, and keeping people at the center of decision-making, we can shape a future where rare disease access is no longer a challenge but a given.
The solutions are within reach. Now is the time to make them a reality.
New in our ‘A Life in Science’ series, we hear from Christelle Huguet, Executive Vice President and Head of Research and Development, about what keeps her motivated in the challenging world of pharma. Driven by a deep sense of curiosity and a strong commitment to advancing patient outcomes, her journey is a powerful testament to the incredible impact of a curious mind and science with a purpose.
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