Lily May, a vibrant and determined seven-year-old, loves gymnastics and golf. Yet, like many children living with pediatric low-grade glioma (pLGG), her life has been profoundly shaped by this rare brain tumor. Although the global burden is unknown1, pLGG is a rare childhood brain tumor, with approximately 700 children diagnosed in the EU5* each year2.
For Lily May’s mother, Lianna, the journey began with unsettling signs.
After months of worry and numerous doctor visits, a diagnosis of optic pathway glioma was made. This type of pLGG, a tumor on the optic nerve, is rare, presenting in approximately 5% of all childhood brain tumors3; Lily May’s doctor even called it a “once-in-a-career find.”
Despite their benign classification, pLGGs are progressive and can cause significant long-term damage as they continue to grow. This reality is common for many children living with pLGG.
Lily May’s tumor has led to near blindness in her left eye, limited peripheral vision and chronic fatigue. Beyond this, pLGG can cause a range of disabilities, including functional complications and neurological symptoms, often leading to developmental delays4.
The journey of managing her condition itself adds to the burden. Lily May’s early years were marked by frequent hospital appointments and medical interventions. When her tumor grew again after two-years, the cycle of intensive care continued. This constant need for medical attention meant Lianna couldn’t return to work, which had a significant impact on their family’s life.
Due to their location, many pLGGs cannot be completely surgically removed5, creating a cycle of treatment and often invasive surgeries. Managing the growth of the tumor together with significant treatment complications throughout childhood is the reality for families, impacting every aspect of children’s development and creating lifelong health issues6.Essential childhood activities such as school, sports, and important social interactions are often missed7 – Lily May’s countless hospital visits have and will continue to have a profound impact on her social life and childhood as a whole.
Psychologically, parents also bear an immense burden, with concerns about the impact of the tumor on their child’s health and well-being, together with the pressure of making critical medical decisions that could impact the future6.
Despite these immense hurdles, children living with pLGG, like Lily May, often show remarkable resilience and are determined to live normal lives. Pursuing her passions and embracing every moment, Lily May’s drive to be like every other child is inspiring, even as she grows to understand that her illness makes her different.
Ipsen is committed to supporting the pLGG community and advancing understanding of this complex condition. We believe that all children deserve the right to a happy and healthy childhood, allowing them to continue doing what makes them happy, and thrive into adulthood.
* France, Spain, UK, Germany, Italy
In a recent conversation, Professor Björn Fischler, paediatric hepatologist at the Karolinska Institute in Sweden, and Laure Dorey, Director of the French patient organisation AMFE and mother of a child living with biliary atresia (BA), together discussed the realities of living with rare liver diseases (RLDs) and the importance of early detection.
RLDs, including progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), and BA, are complex and chronic conditions that can profoundly impact children and their families.
Often in RLDs, the flow of bile from the liver to the digestive tract is disrupted, a condition known as cholestasis. When bile becomes trapped in the liver, it causes inflammation, which can lead to scarring and may be associated with a gradual decline in liver function.i Bile acids, which are a key component of bile, can accumulate in the blood, enter the skin and irritate nerve endings.ii,iii
One of the challenges in diagnosing RLDs is that early symptoms can be subtle or misleading. Signs such as pale stools, jaundice (yellow skin and eyes), and pruritus (itching), are often overlooked or mistaken for more common childhood conditions.iv
“The most important signs in the very young child would be jaundice… They could also be in a slightly older infant itch or pruritus.” – Professor Björn Fischler
In BA, timing is crucial. Surgery to reroute the flow of bile in the liver within the first weeks of life can significantly improve outcomes.v In PFIC early diagnosis allows for more personalised care plans that help to relieve debilitating symptoms of itch and can preserve liver function.vi
The impact of RLDs goes beyond physical health. Children experiencing intense pruritus often struggle with sleep, appetite, growth, and energy levels.vii “They don’t sleep at night, maybe they don’t eat properly, and maybe they don’t grow properly,” explains Professor Fischler. “So they tend to be a bit weaker and a bit less motivated to do different activities.”
These challenges affect the wider family too. Parents often face emotional stress, sleep deprivation, and the constant demands of care. “It is actually detrimental for the whole family system,” he adds.
Supporting families with clear, reliable information and consistent care is essential. RLDs are complex, meaning caregivers need to become well-informed and proactive to advocate effectively for their child’s needs. Access to trusted information and open communication helps families feel more confident and better equipped to manage care.
“Parents need to feel that they are listened to, and they need to be supported,” – Laure Dorey.
Ipsen is committed to improving outcomes for people impacted by rare liver diseases. By working together with the community, we gain a deeper understanding of what matters most to them and how to best meet their needs. Through this collaboration, we strive to improve lives and transform the outlook for those living with rare liver disease.
Professor Björn Fischler and Laure Dorey were compensated by Ipsen for their time.
“Fatigue is so different to being tired, I can’t get out of bed… it is life changing.”
– Wendy, living with PBC
PBC is a rare, progressive, autoimmune, cholestatic liver disease with prevalence increasing globally 3
Autoimmune means that in people with PBC, a type of white blood cell found in the body attacks and gradually destroys the liver’s small bile ducts3
Bile ducts are tiny tubes in the liver that help carry bile, a fluid containing bile acids which breaks down fats and helps remove waste from the body4
When the bile ducts are damaged, bile can’t flow properly and starts to build up in the liver which causes stress and damage to liver cells3
Over time, the liver gets scarred (fibrosis), and if it keeps getting worse, it can lead to cirrhosis (serious liver damage) and even liver failure3
Up to 20% are experiencing severe fatigue
Fatigue is not simply a secondary effect of other symptoms such as pruritus.
While pruritus can contribute to the experience of fatigue, research shows the two symptoms are only weakly correlated,7 indicating separate mechanisms are involved.
This suggests that fatigue can improve independently with targeted intervention, reinforcing its status as a treatable condition.
Fatigue is a clinically significant condition in PBC
Studies show up to 37% of people with PBC continue to experience fatigue post-liver transplant, making it a condition that requires its own focus.9
*Severe fatigue refers to a significant cause of quality of life impairment11
**Fatigue is assessed using validated patient-reported outcome tools1
While fatigue is common among people living with PBC, there is now evidence that some treatments for PBC may improve fatigue.7,10
Advanced brain imaging techniques are showing changes in brain activity and connectivity associated with fatigue in people living with PBC.12
Recent research suggests that fatigue in PBC is linked to disrupted communication between brain areas involved in motor control, sensory processing and emotional regulation.12
Fatigue can have a range of physical, emotional, and cognitive impacts that can deeply disrupt quality of life
Limits movement and daily function13
Disrupts sleep quality, memory and focus14
Anxiety, depression, isolation15
Strained relationships and difficulty maintaining employment16
Up to 55% of people living with PBC with severe fatigue experience significant cognitive symptoms.14
Fewer than 20% people living with PBC with severe fatigue are able to work.16
“Sometimes it’s difficult to get through a day at work… and then it impacts your social life… even speaking can be an effort when you’re struggling with fatigue.”
– Jo, living with PBC
Fatigue is not just a symptom – it’s an all-encompassing and far-reaching burden that has ripple effects that extend far beyond the individual.
It often shifts daily responsibilities onto family and caregivers, leading to emotional distress and financial strain.17,18
It can also contribute to reduced workforce participation and early retirement.19,20
Up to 60% of people with PBC report a loss in work productivity due to the disease.19
As symptom impact progresses, many people are unable to afford the care they need for their long-term, or chronic conditions, such as PBC. This in turn may result in friends and family providing free care which could lead to loss of work and wages.17,18
Despite its prevalence and impact, fatigue remains undermanaged and often overlooked in clinical care.8
While validated tools and clinical thresholds exist to track fatigue, they are not consistently implemented in practice,21 resulting in missed opportunities for management.
There is a critical need for improved recognition, routine measurement and open communication around fatigue in PBC, not just as a symptom, but as a core condition that significantly impacts quality of life.
Treatment must go beyond liver biochemistry and address both disease progression and the burden of symptoms, like fatigue – healthcare professionals should see and treat the whole person, not just the liver disease.
Ipsen’s Sandra Silvestri sat down with Dr. Laura Cristoferi, Professor Mark Swain, and Professor David Jones to explore the latest evidence on management of the rare, autoimmune disease, primary biliary cholangitis (PBC). In the discussion, the panel highlighted the importance of holistic, individualized care approaches and empowering patients to take an active role in managing their condition. They also discussed the need to address symptoms that matter most to patients to improve outcomes and overall well-being.
Every person’s journey with PBC is unique. While slowing disease progression is a priority, management of symptoms is also huge priority for those living with PBC, as these are often the aspects that impact their life the most. Two of the most common symptoms that impact people living with PBC are fatigue and pruritus (itching).
Fatigue affects up to 80% of people living with PBC,1 with 20% reporting it as significant or life-altering.2 It can significantly impair daily functioning, limit physical activity, and affect emotional well-being, independent of pruritus. It therefore requires specific recognition and management as a distinct symptom of PBC.
“It is refreshing to see where the field has moved – to the point where we’re not only talking about ‘Are they able to live a long life with their disease?’, but ‘Are they actually living a good life while they live that long life?’”– Mark Swain, hepatologist at the University of Calgary in Calgary, Canada
Growth in the scientific understanding of the symptoms of PBC and the biology behind them has led to the recognition that fatigue, once simply labelled as being ‘tired’, may be linked to a biological pathway. As we start to understand the full spectrum of symptoms and the mechanisms driving them, management of PBC can become more informed and tailored to specific needs.
Enhancing patient understanding of PBC is essential, and healthcare professionals should take the time to explain key aspects of PBC, such as alkaline phosphatase levels. Just as importantly, they should actively listen to and validate the experiences of those living with PBC. Together, these actions help people living with PBC be more empowered to take an active role in their care and work alongside their healthcare team to manage the PBC they live with.
Our understanding of the heterogeneity of PBC is growing, and it’s clear that managing the condition requires more than routine monitoring. It demands a personalized, holistic approach that looks beyond lab results and focuses on the symptoms that matter most to each individual living with PBC.
The first six months of 2025 have been exceptional for Ipsen. We’ve delivered strong growth across all three of our therapeutic areas — Rare Disease, Oncology, and Neuroscience — reflecting the strength of our strategy and the dedication of our teams.
We’ve also made remarkable progress in our pipeline and portfolio, with new approvals and promising assets advancing into clinical development. These milestones are a testament to our continued focus on innovation and patient impact.
Looking ahead, the second half of the year promises even more exciting developments. We’re on track to reach key milestones that will further strengthen our position and bring new hope to patients around the world.
At Ipsen, we’re not just executing on our strategy — we’re transforming lives. Because we believe that everyone deserves a life fully lived.
Joris is a thriving seven-and-a-half-year-old boy who has been on a journey requiring strength and hope from himself, and all those around him. At just four weeks old, Joris began exhibiting unusual symptoms; vomiting, changes in his eating habits and he had very pale poo. Concerned, his mother, Maria, took him to a pediatric doctor. Unable to pinpoint the issue, they were referred to a liver specialist hospital. A blood test revealed irregular liver values, prompting an urgent stay for further diagnosis.
Doctors suspected biliary atresia. This is a rare liver disease affecting infants that causes a blockage in the bile ducts in the liver. Symptoms include jaundice, hardened liver, swollen abdomen, pale stools and dark urine, which persist beyond the first two weeks of life1.
A small exploratory surgery was performed, and while Joris’ liver appeared healthy, concerns remained.
“I felt quite anxious and didn’t really know what was happening. That was quite challenging as a mother.”
At three months old, Joris was officially diagnosed following a crucial test, which confirmed biliary atresia. At this age his doctors advised he was too old for Kasai surgery, which re-routes bile away from the liver. His only chance of survival was a liver transplant.
Maria and her husband explored the possibility of becoming living donors themselves and testing confirmed Maria as a match.
When Joris’ condition deteriorated, they made the difficult but necessary decision to proceed with the transplant. On December 4th, the surgery took place successfully. Challenges followed—his gallbladder was removed, and his bile ducts adjusted, leading to post-surgical complications. Drainage tubes supported his recovery for a year, but ultimately, he healed.
His scars are a testament to his resilience, unnoticed by him as he embraces life with enthusiasm. He excels in school, enjoys swimming, and has recently discovered a passion for baseball. His journey, marked by uncertainty and perseverance, has shaped him into a strong and determined child.
Maria reflects on their experience with gratitude for medical advancements, family support, and the ability to be informed. Through research, she gained understanding—knowledge that lessened the shock and enabled her to make informed decisions with confidence.
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An investor and analyst conference call will follow at 13:00 CET.
31 July 2025 – 1 mins read
“Our half year results reflect continued strong momentum, with growth across all three therapeutic areas, particularly in our rare liver disease franchise, which is expanding rapidly and progressing well,” said David Loew, Chief Executive Officer, Ipsen. “Building on that performance,…
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.
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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.
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