Every person’s experience living with neuroendocrine tumors (NETs) is unique and for Melanie, coming to terms with a diagnosis of lung NETs is an ongoing journey, building knowledge and understanding of how to live with this complex and often unknown form of cancer.

Getting a diagnosis

As is so often the case for many people, Melanie’s diagnosis came by chance following a routine check-up for a cough. When a subsequent x-ray revealed some shadowing on her lungs, her GP initially diagnosed her with lung cancer, with a very poor prognosis, something she wasn’t told in the appointment, but discovered in her notes. This caused immense distress at the thought of what this would mean for her future. However, it was only after a biopsy was conducted that the true nature of her diagnosis was revealed: Melanie was in fact living with an advanced form of lung NETs, a term she had never heard of before.

The quest for knowledge

NETs are a relatively uncommon form of cancer that affects the cells of your neuroendocrine system.¹ These cells are found throughout your body, but NETs most often occur in the lungs, stomach, or pancreas.² Most forms of NETs grow slowly and are less aggressive,³ a point the doctor was keen to emphasize to Melanie. However, being diagnosed with cancer of any form is life changing.

While Melanie finally had an answer, the uncommon nature of NETs, especially lung NETs, meant that information about the disease was scarce. Yet, with approximately 35 in every 100,000 people worldwide living with NETs,¹ and lung NETs accounting for 27% of all cases,² this type of cancer is far more prevalent than many realize. In order to seek out more information, Melanie sought a second opinion from a professor at a NETs Center of Excellence, who had the expertise to discuss the complexities of her diagnosis.

While Melanie was reassured that her NETs could be treated, it wasn’t operable. This meant she had to learn to live with it, which is a continually evolving process – and to do so, she had to gain a deeper understanding of this complex disease. Melanie credits a national patient advocacy group for being her crucial support network, offering understanding from others facing similar journeys and providing a hub of information to turn to.

“Everyone is completely and utterly different. But at the same time, incredibly supportive and positive and, even on the really hard days, there is love and you feel like you’re part of a big family.”

Being seen and understood

Having an uncommon cancer can feel isolating. While Melanie feels physically well most of the time, she still shoulders the burden of living with a form of cancer where care options are difficult to navigate and the future is unknown. Recently, Melanie attended appointments with a friend as they were diagnosed with a more common type of cancer highlighting the disparity in care between cancer types. The medical knowledge, funding and specialist support experienced by those with less common cancers can be stark; from a lack of dedicated nursing staff, timely scan results, patient information, support group awareness and even funded parking at hospital car parks.

A long-term outlook on life

Melanie has accepted that her NETs may grow, change, shrink, or spread, but she is still determined to live well; enjoying activities for what they are, not just associating them with providing relief from her diagnosis. Her job as a gardener and recently discovered love of sea swimming means she can continue to enjoy her passion for the outdoors. Learning to live with NETs is an evolving journey, one Melanie is taking one step at a time.

Ipsen Q3 Results Call

The investor and analyst conference call will take place on 22 October 2025 at 1pm CET. For the Q&A (audio only) please join here.

Register

Speakers

David Loew

Chief Executive Officer

Aymeric Le Chatelier

Executive Vice President

Chief Financial Officer

Christelle Huguet

Executive Vice President

Head of R&D

If you are experiencing difficulties registering for the investor and analyst conference call, please contact: khalid.deojee@ipsen.com

Hugo Gomes da Silva, SVP Global Medical Affairs (Rare Disease) at Ipsen, shares powerful insights into the evolving challenges families face throughout their PFIC journey—and highlights the critical importance of trusted information and community support.  

As we mark PFIC Awareness Day on October 5, I reflect on what I have heard from those living with Progressive Familial Intrahepatic Cholestasis (PFIC) and how the disease impacts them not just on one day, but every single day. PFIC is a rare genetic liver disease, often diagnosed in infancy or childhood, that can cause progressive liver damage, severe itching, and has a profound impact on quality of life. 

This year’s theme, Stronger Together, speaks to the power of unity, empathy, and shared knowledge within this community. For families, this theme represents not only awareness of the condition itself, but also the reassurance that no one should face PFIC alone. 

I have had conversations with those parents who embody this spirit of strength and advocacy. One of them is Emily Ventura, co-founder of the PFIC Network and mother to Cedar, who lives with PFIC. She remembers how difficult it was in the early days of her daughter’s diagnosis: “Learning what PFIC really meant for my daughter was overwhelming. I felt lost and unsure of where to find reliable guidance.”  

What helped Emily most was finding a community of families who had walked the same path, transforming her fear into confidence. Inspired by this, she went on to co-found the PFIC Network, which provides both trustworthy information and a safe space for families to connect. 

Francesca faced a similar journey when her daughter, Eva Luna, was diagnosed with PFIC as an infant. The first months were filled with uncertainty and fear as they tried to understand what the diagnosis meant. One of the most difficult challenges was managing Eva Luna’s persistent itch, which Francesca described as “like a monster” that disrupted family life. Helping her daughter feel comfortable and live a happy, active childhood became the focus of their journey. Many other families share this challenge of balancing symptom management with daily life and hope for the future. 

For Francesca, connecting with other mothers gave her hope and perspective: “It was very important to me to be in touch with the other mothers around the world. It gave me a lot of hope, because I saw their children grow, go to school and even live a normal life.” This led her to set up PFIC Italia, which not only supports those in Italy, but has international reach.  

Emily’s and Francesca’s journeys show us that behind every diagnosis is a family searching for clarity, support, and the reassurance of not being alone. To navigate PFIC, families need both knowledge that empowers them and a community that gives them strength. 

At Ipsen, my colleagues and I are committed to listening to families and standing alongside them. PFIC Awareness Day reminds us that advancing science is only part of the solution; solidarity and support are equally vital. By standing stronger together, we can help families feel informed, empowered, and never alone on their journey. The PFIC Network is a trusted source for families, bringing together the full spectrum of information, tips, and real experiences in one place. 

To learn more about PFIC, visit the PFIC Network’s website: PFIC.org and the PFIC Italia Network 

“We collaborate with our medical and chemical colleagues doing drug discovery, taking a look at the current process, and thinking about the best ways to improve efficiency and minimising environmental impact.”

Nathan McCormack focuses on the earliest stages of active pharmaceutical ingredient (API) development. This involves working alongside medicinal chemistry colleagues to identify new molecules, then working to make them practical for scale-up and manufacturing. Part of the API team in Dublin, Nathan supports new molecules for the three Ipsen therapeutic areas of neuroscience, rare disease, and oncology, partnering closely with medicinal chemistry colleagues to assess how a compound is made—and how that process will hold up under large-scale constraints. “What you use on a small scale may not necessarily translate,” he says. “In terms of safety, it’s something we have to think about on a manufacturing scale.”

Nathan’s job is not just about synthesis. He and his colleagues evaluate the feasibility of every reaction route, identifying risks and opportunities early. “We’ll support the supply of this API through preclinical and clinical stages,” he explains, “until we reach commercial manufacture.”

That role also includes tracking what happens to a compound after it enters the body. “We work closely with our external partners,” says Nathan. These partnerships play a large role in innovation and building better processes for sustainability. What excites Nathan most is driving to improve sustainability and environmental footprint. Ipsen is a member of the American Chemical Society Green Chemistry Institute Pharmaceutical Roundtable (ACS GCI PR), a collaboration with other pharmaceutical companies that Nathan and his coworkers see as critical. “We’re already taking action—benchmarking, analyzing, and changing what we do.” This is done to reduce environmental impact while maximizing scientific discovery.

That work also includes partnerships with academic labs. “They come up with some of the best innovations,” he says. “And we can help them adapt it for industrial applications.” For Nathan, sustainability is not a side project—it is the future of drug development.

Naomi Binoche, VP Head of Strategic Alliance Management, shares insights on how pharma partnerships are key to driving success and why celebrating every milestone along the way matters.

This theme was further explored at the ASAP Global Alliance Summit in Amsterdam recently, where Naomi co-presented the session “Living and Growing an Inherited Alliance – a Journey full of Twists and Turns.” The presentation highlighted how Ipsen and Servier overcame the challenges of an inherited partnership to build a successful alliance – a journey that reflected the resilience and adaptability required in the pharma landscape.

“For me, success is not just about big breakthroughs. In nearly 10 years with Ipsen, I’ve learned it’s just as much about building trusted partnerships and celebrating every milestone along the way. We thrive by working together – it’s what drives innovation across our therapeutic areas and ultimately helps us deliver transformational medicines to patients worldwide.

Moving forward together can be transformational. When combining Ipsen’s global reach, size, and expertise with our partners’ cutting-edge research we are able to consistently push new and exciting programs through the pipeline. I’ve been very fortunate to contribute to many successful partnerships that have advanced our programs; moving them from pre-clinical development, through regulatory approval, and finally into the market where they can benefit those in need.

What fulfils me every day is the diversity of partners we work with: from the US-listed biotech to the family-owned business in Europe; the ambitious young enterprise in China, to the established industry leader. Each brings its own unique challenges and opportunities, and it is this variety that makes my day – every single day!

Turning a molecule into a medicine is a long and complex journey, demanding years of dedication and collaboration. That’s why it’s so important to pause and celebrate every win along the way. These shared milestones – whether operational, strategic, or relational – are the heartbeat of alliance success, helping to build trust and momentum over time.

An alliance isn’t just about the initial collaboration or license agreement. It’s about all the supporting pieces that go alongside. Pharmacovigilance, quality, and data protection, manufacturing, supply, and life-cycle opportunities and more. Each achievement is something we secure together with our partners over time. These milestones might not make the headlines, but for me and my team, they’re standout moments that reflect the strength of our collaboration.

Of course, the journey doesn’t stop after bringing a medicine to market. Keeping our medicines available in over 100 countries takes enormous effort, with teams around the world working together every day to make manufacturing and distribution happen. This is where our long-term alliances really shine. Some of our partnerships span over 30 years, with off-patent molecules still achieving double-digit growth in certain regions.

Celebrating each milestone with our partners, especially during what can be a long and challenging journey, fuels our motivation to push programs forward and ensure medicines remain accessible. In the end, making a real impact for patients is what matters — and that’s always worth celebrating.”

An article by Josep Catllà, Executive Vice President, Chief Corporate Affairs Officer

For the thousands of individuals, mostly women, living with primary biliary cholangitis (PBC), a chronic, progressive autoimmune liver disease, the journey can be lonely – not just because of the disease itself but because it is often misunderstood, minimized, and overlooked.¹ 

Liver disease carries a lot of stigma, mainly because of the assumption that it’s caused by lifestyle choices. However, many liver conditions, such as PBC, aren’t related to behavior and don’t produce visible symptoms. Instead, PBC brings debilitating fatigue, itching, and brain fog – symptoms that are invisible to others, yet deeply disruptive to daily life.¹ 

Liver disease is too often met with judgement and with women making up 9 out of 10 people living with PBC², their symptoms are even more likely to be dismissed. A survey conducted by EURORDIS found that women with rare diseases face longer diagnostic journeys than men, even when comparing conditions equally prevalent in both sexes.³ 

Women’s discomfort and instincts about their health have historically been minimized. Fatigue, for example, is socially normalized in women due to societal roles (work, caregiving, household responsibilities), and studies show that it is more likely to be dismissed as psychological, hormonal or simply ‘normal’ – even when it signals a serious underlying condition.^4,5 

Middle-aged women, in particular, are at risk of experiencing fatigue from multiple overlapping sources, such as menopause, which makes it harder to reach a clear diagnosis or get the right support and treatment.⁶ Since PBC is most commonly diagnosed in women between the ages of 35-55⁷, symptoms such as fatigue and brain fog are often misattributed, making timely recognition and appropriate care more challenging.⁵ 

This lack of recognition can have a serious knock-on effect on mental wellbeing. Many people living with PBC describe a sense of disconnection – not just from their health, but from their communities, workplaces, and support systems.⁸ That’s why recognition, awareness, and validation of PBC is essential in shortening time to diagnoses, improving disease management, and personalized care.  

Thanks to advocacy, research, and the patient support networks available, more people are listening to women’s health concerns and recognizing that symptoms, like fatigue, are real. When patients feel dismissed or isolated, support groups can become lifelines. They remind people with PBC that they’re not alone and that their experiences matter. This is crucial to addressing the broader issue of gender disparities in healthcare, and especially important for women with rare diseases, who face additional barriers to diagnosis and care.  

Improving recognition, reassurance and support is important across all rare diseases – especially for individuals who may feel disengaged from their condition, who may not be members of support organizations, or who do not feel like they fully understand how to manage it. This is especially important in PBC, and our recently launched campaign, This is My PBC, aims to provide individuals with recognition, reassurance, and validation in how they experience their disease, and empowers those living with PBC to take charge of their journey. People living with PBC may be unique, but they are not alone. 

To learn more about PBC and fatigue, visit:Beyond tired: Understanding fatigue in Primary Biliary Cholangitis – Global

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.

“I knew there was something not right from about four weeks old because Lily May spent many hours crying, but she sounded like she was in pain.” – Lianna

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.” 

The Hidden Burden of Benign Tumors

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.

“Your idea of life is completely turned on its head and you want to protect your child and you’re going to do what it takes for them to survive.” – Lianna

Resilience Amidst Challenges

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.  

Understanding fatigue

• Fatigue is a distinct and debilitating condition that can have a significant impact on quality of life¹
• It often accompanies serious autoimmune diseases such as primary biliary cholangitis (PBC), lupus, multiple sclerosis, and other chronic inflammatory conditions²
• While tiredness can often be relieved by rest or sleep, fatigue is a chronic condition that isn’t improved by rest¹
• Fatigue is persistent, overwhelming and not proportional to activity. It affects physical, emotional, and mental functioning, often interfering with daily life² 

About 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

Most common symptoms of PBC

Fatigue

Pruritus (itching)

Fatigue is a common and debilitating symptom of PBC

Fatigue affects up to 80% of people with PBC

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 measurable

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

Uncovering the science of fatigue in PBC

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

A daily struggle that touches every part of life

Fatigue can have a range of physical, emotional, and cognitive impacts that can deeply disrupt quality of life

Physical impact 

Limits movement and daily function¹³

Cognitive impact

Disrupts sleep quality, memory and focus¹⁴

Emotional impact

Anxiety, depression, isolation¹⁵

Social impact

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

Far reaching impact of fatigue

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

60% of people with PBC report a loss in work productivity

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

Fatigue remains unaddressed

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.

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.