• A hidden diagnosis: Although PFIC is widely considered a childhood condition, some people do not experience recognizable symptoms until adulthood, leaving many undiagnosed or misdiagnosed for years 

• The cost of missed recognition: While adults search for answers, liver damage may continue silently progressing, alongside the physical and emotional impact of symptoms such as itching, fatigue and uncertainty 

• No one should navigate PFIC alone: Through initiatives like PFIC Colors, developed with the PFIC Network, we are helping people better understand PFIC, connect with trusted support and feel empowered to seek answers and specialist care 

What is adult PFIC? 

PFIC is a group of rare inherited liver conditions that affects the body’s ability to transport and circulate bile, which is essential for digesting and absorbing fats, as well as removing waste products.^1–5 When bile builds up in the liver , it can damage the liver and cause symptoms such as itching.¹ 

While PFIC is often associated with childhood, it is increasingly recognized that it can also manifest in adults.^3,6 In these cases, symptoms may not appear until later in life, sometimes triggered by events such as pregnancy or an infection.^2,6 

For many, this means years of unexplained symptoms, repeated medical appointments and uncertainty before the underlying cause is identified.⁶ 

What is the impact of PFIC? 

For adults living with PFIC, the condition can affect nearly every aspect of life, from physical health and emotional wellbeing to work, relationships and future plans.⁶ 

Common symptoms include itching (pruritus), yellowing of the skin and eyes (jaundice), abdominal pain, diarrhea and fatigue, which can disrupt everyday activities and quality of life.^7,8 

For many, the most challenging symptom is the relentless, debilitating itching. Because itching has no visible signs, its severity is often underestimated, making it difficult for others to understand the exhausting and all-consuming burden, which can extend far beyond physical symptoms.^1,6,9,10 

Severe itching can affect concentration, work, education, family life, relationships and mental wellbeing. Sleep disruption caused by persistent itching can leave people exhausted during the day, while the unpredictability of symptoms and their underlying cause may also create anxiety about the future.^1,10–13   

Many people describe feeling isolated, particularly because PFIC is rare and poorly understood by others.⁶ 

Moreover, PFIC is a progressive condition, which means it can evolve over time. One of the challenges of the disease is that underlying changes in the liver may continue even when symptoms fluctuate or seem manageable. As a result, individuals may feel relatively well while the disease remains active beneath the surface.⁶ 

Without appropriate care, bile continues to build up in the liver, which can cause long-term damage and may eventually lead to liver failure.^1,6,14 

Why is a diagnosis of adult PFIC so challenging?  

Adult PFIC is often misdiagnosed or diagnosed much later than it should be. This is primarily driven by limited awareness and understanding in adults.^6,13 The long-held perception of PFIC as a childhood condition can mean healthcare professionals (HCPs) may not immediately suspect it in adults.^6,14 

Contributing to this challenge is that the condition rarely presents in a straightforward or predictable way. Some adults may have mild or no obvious symptoms at all.^6,15 For those who do experience symptoms, they often overlap with those of other, more common conditions.⁶ In some cases, symptoms come and go over time,⁶ making it difficult to identify the underlying cause.  

As a result, PFIC is often overlooked, dismissed or attributed to other causes,^6,13 which can leave people feeling frustrated and unsupported. Indeed, many adults with PFIC spend years, or even decades, searching for answers, often only managing symptoms without optimal care – all the while, liver damage may continue to progress.⁶ 

How can we bridge the diagnostic gap in PFIC? 

Earlier recognition is essential to changing the disease trajectory and improving long-term outcomes for adults with PFIC.^16,17 The first step is increasing awareness of adult PFIC, so that symptoms are recognized in this context sooner and appropriate testing can take place.⁶ 

When PFIC is suspected, genetic testing is often used to confirm the diagnosis.^16,17 Because the condition is caused by changes in certain genes, genetic testing can identify the underlying cause. However, genetic variants are not always found.^18,19 In some cases, a diagnosis is ultimately made through a comprehensive clinical assessment and the exclusion of all other possible causes.^18,19 

A confirmed diagnosis can be an important turning point 

For some people, finally receiving a diagnosis provides a long-awaited validation that their symptoms have a genuine underlying cause. It can also open the door to specialized care and access to a supportive community.¹⁷ 

When should PFIC be considered? 

Could PFIC be the missing explanation? Adults with the following symptoms or experiences may benefit from discussion with a liver specialist:^3,6,17,19 

• Persistent or recurrent unexplained itching 

• Cholestasis or unexplained abnormalities in liver tests 

• Episodes of jaundice 

• Family history of liver disease 

• Symptoms that have remained unexplained despite previous investigations 

• Ongoing liver problems with no clear cause 

• History of severe or recurrent intrahepatic cholestasis of pregnancy (ICP) 

Please note: This information is intended for educational purposes only and is not a substitute for medical advice, diagnosis, or treatment. Please speak with your doctor or healthcare team if you have questions or concerns about your health or the possibility of PFIC.  

Aurora Berra, Vice President Global Patient Officer

We are living in an era of extraordinary scientific progress. We can target diseases in ways that were unimaginable a generation ago, develop increasingly sophisticated medicines and explore entirely new approaches to treatment.

Yet there is a paradox at the heart of modern healthcare: as science becomes more advanced, understanding it can become more difficult. That is why health literacy matters. A breakthrough can only reach its full potential when the people it is intended to help can understand what it could mean for their lives.

At Ipsen, much of our focus is in areas where significant unmet need remains and patients are often waiting for meaningful advances. We brought forward the first-ever approved treatment for fibrodysplasia ossificans progressiva (FOP), helped expand options for people living with primary biliary cholangitis (PBC) and are advancing targeted treatment approaches to children with pediatric low-grade glioma (pLGG).

For the people and families affected by these conditions, scientific progress is not an abstract concept. It can bring new possibilities where options have been limited. With every advance comes a responsibility, not only to make the science a reality, but to bring patients and communities on the journey with us.

Effective communication is often one of the hardest parts of that journey to get right. Health literacy is varied and nuanced. It is often framed around a patient’s ability to understand complex information. But I believe we need to ask a different question: have we communicated the science clearly enough?

That shift matters. It places the responsibility on those of us creating and sharing health information. Our role is not simply to provide facts, but to make them genuinely useful, so people can ask the questions that matter to them and make informed decisions about their care. That means challenging ourselves at every step, thinking creatively and being open about what we know, as well as where uncertainty remains.

Clarity must start at the outset, not once the science is complete and the materials are written. Some of our most valuable teachers are patients and caregivers themselves.  Our Patient Expert Review Panels help ensure materials reflect the realities of living with a condition, challenging assumptions and uncovering barriers we might otherwise miss.

Our Plain Language Review Panel brings another perspective, helping us identify language or concepts that may not be as accessible as intended. We aim to apply these learnings across everything we share, from visual explainers and plain-language summaries to open access to our published research.¹ Each is part of the same ambition: to remove barriers between scientific progress and the people it is intended to serve.

Science will continue to move at pace, particularly in the areas we are pursuing at Ipsen. How we communicate must evolve alongside it.

Because innovation is not only about discovering what is possible. It is about ensuring people can make sense of what is possible for them. For me, that is what responsible innovation looks like.

Science is at the heart of everything we do. It also comes with an environmental footprint, from the energy and water used in laboratories to the chemicals, equipment and materials required to advance our work. 

As part of Ipsen’s sustainability strategy, we are taking practical steps to reduce that footprint and preserve vital resources. At our Dublin and Wrexham sites, we are putting this ambition into action through My Green Lab Certification, a globally recognized program designed to help laboratories embed sustainability into their everyday operations. 

A global framework for more sustainable science 

My Green Lab Certification provides laboratories with a structured approach to assess their environmental performance, identify opportunities for improvement, and turn sustainability ambitions into practical action. 

Today, more than 4,000 laboratories and 50,000 scientists across over 50 countries have participated in My Green Lab Certification. The program is endorsed by the UN-backed Race to Zero and recommended by the U.S. Environmental Protection Agency (EPA). 

The certification addresses 14 areas of laboratory sustainability, including energy, water, waste, chemicals and materials, procurement, and employee engagement. Laboratories establish a baseline, implement improvements, and measure their progress before completing the certification assessment, with certification results independently verified. 

Putting the framework into action at Ipsen 

Teams in Dublin and Wrexham are applying the My Green Lab framework across laboratories with different activities and ways of working, creating an opportunity to test approaches, share learnings and identify practical solutions.

The work starts by looking closely at everyday laboratory practices. 

In Dublin’s APID laboratory, for example, an energy audit estimated that the equipment assessed uses approximately 94,220 kWh of electricity each year. The findings helped the team identify concrete opportunities to reduce energy use, from putting a GCMS (used to analyze chemical sample) instrument into sleep mode to powering down equipment when it is not needed. 

Other actions underway include: 

• improving chemical inventory management and sharing chemicals between departments to help prevent unnecessary waste; 

• introducing reusable alternatives to selected single-use laboratory items; 

• installing low-flow aerators to reduce water consumption; 

• consolidating shipments where possible; and 

• introducing Sustainability Champions and dedicated sustainability training to help embed new practices. 

Building a culture of sustainable science 

Making laboratories more sustainable is not only about equipment and resources. It also requires people to look differently at how they work. 

Sustainability Champions and lab leads are helping drive implementation within their teams, while colleagues in Dublin and Wrexham meet regularly to compare progress, share experiences and learn from one another. 

This cross-site collaboration is helping teams explore what works across different laboratory environments while building sustainability into everyday scientific practices. 

Working toward certification 

Dublin and Wrexham will continue implementing improvements as they work toward My Green Lab Certification that will provide external recognition of the sustainable practices adopted by the participating laboratories and a framework for continued improvement. My Green Lab certification is valid for two years, reinforcing the importance of continuing to assess, improve and embed sustainable practices over time. 

For Ipsen, the value of this first phase goes beyond certification. By testing, measuring and sharing what works in Dublin and Wrexham, we are building practical experience that can help us advance more sustainable laboratory practices across Ipsen.  

Behind every job title is a human story

Nobody arrives at work as just an employee. Behind every scientist, every assistant, every account manager, there’s always someone else. A patient. A caregiver. A parent. A child. And that changes everything.

Meet Carole, Agnès, Ed and Jacquie

They work at Ipsen, but their job titles tell only part of who they are.

We asked them to share the experiences that have shaped their lives and perspectives, and to tell us more about who they are beyond work.

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Carole

When Carole’s mother was diagnosed with Alzheimer’s, her whole family was affected. Over the years, the experience has profoundly changed the relationship between mother and daughter.

Agnès

When both of Agnès’ parents were diagnosed with cancer within months of each other, she found herself facing the possibility of losing them both. As a daughter, mother and caregiver, the experience changed the way she thinks about caring for others and herself.

Ed

Ed was diagnosed with kidney cancer when his daughter was just six months old. Today, as a cancer survivor and Oncology Account Manager, his personal and professional worlds intersect in a way he never expected.

Jacquie

A mother and a scientist, Jacquie has been living with axial spondyloarthritis for over a decade. Living with a chronic disease meant adjusting to a new reality. It also brought a deeply personal perspective to her work.

There’s always more behind the job title.

Follow #ThisIsIpsen and share the stories that resonate with you.

Ipsen’s commitment to developing strong capabilities across its teams has been recognized with two Gold Awards at the 2026 Brandon Hall Excellence Awards, one of the leading international programs recognizing achievements in learning and talent development.

The awards recognize two initiatives supporting the development of Ipsen’s commercial teams:

• Gold | Best Use of AI for Learning
  DARWIN, Ipsen’s AI-powered role-play platform, enables sales representatives to practice real-world conversations with healthcare professionals in an interactive and realistic environment.
• Gold | Best Competencies & Skills Development Program
  Ipsen’s First-Line Sales Manager (FLSM) Coaching Programs help sales managers strengthen their coaching capabilities and support the development of their teams.

Together, these award-winning initiatives reflect Ipsen’s commitment to investing in the growth and success of its field teams. The recognition reinforces the importance of ongoing learning and development opportunities that support professional growth, career progression and long-term success for sales representatives and managers.

The value of these initiatives goes beyond industry recognition. By equipping teams with the skills, knowledge and confidence to excel in their roles, they contribute to our ability to better support healthcare professionals and, ultimately, patients.

“In order to help all partners understand the value of a medicine, we provide the wrap-around support of data coming out of clinical trials.” 

Clinical trials answer specific questions in very structured settings and under controlled conditions. Seema Meloni’s team ensures those answers exist within a broader context.  

Her focus is on real-world evidence strategy: defining unmet needs, quantifying disease prevalence, and understanding “what the world looks like with or without our therapies.”  

She works across asset teams to build a comprehensive evidence package alongside the clinical development plan. Clinical trials show efficacy and safety under controlled conditions. Real-world evidence complements trials by capturing the full patient experience and how a medicine performs across broader populations. Her team generates additional evidence to answer those questions and place trial results into context. 

“One of the biggest challenges in pharma is external acceptance of real-world data,” she says. “In these cases, we have less control over data collection and the quality of what is collected compared to traditional randomized control trials, so we have to work hard to ensure we introduce strategies to find the highest quality data and analyze them in the most robust way.” 

Randomized trials remain essential. But she argues that in many instances they are not sufficient on their own to support all scientific and business needs. “We often need additional supportive data to complete the full package of understanding the benefit-risk profile of a medicine,” she says. That requires studies designed with scientific rigor while providing broader data sources to various key external stakeholders.  

“The risk is that without a comprehensive and feasible evidence strategy, treatments that work won’t reach the patients who can benefit from them or can sometimes get removed from the market,” she notes. The consequence is not abstract. It affects patients who may rely on those medicines. 

Her conviction was shaped early in her career. After completing her doctorate, she joined an effort to scale HIV treatment across multiple African countries. “It was trial by fire,” she says. She built electronic medical records systems to capture data at scale, learning how evidence informs patient access, ethics, and policy.  

Today, that perspective remains central. “Science with Purpose is making sure we are being responsible and making decisions based on comprehensive, high-quality data,” she says. 

Evidence is integral. It is protection for patients, for decisions, and for the future of medicines. 

After 25 years working in oncology, Jon Travers understood the impact cancer can have on patients and their families. Throughout his career, he had been driven by the possibility of helping bring new treatments to the people who need them most. 

But around ten years ago, that mission became deeply personal. 

When Jon’s mother was diagnosed with a recurrence of melanoma, it was the third time she had faced cancer. Nearly two decades after her previous diagnosis, the disease had returned, and the outlook was poor. Like many families facing a cancer diagnosis, they were confronted with the possibility that they might have very little time left together. 

An opportunity to access an innovative treatment through an expanded access program changed the course of that journey. 

The treatment gave Jon’s mother an additional year of life and, most importantly, an additional year of quality life. It gave the family time to be together, create memories, and share moments they might otherwise never have had. 

Witnessing that impact firsthand changed the way Jon viewed his work. What had once been a professional commitment to innovation became a personal understanding of what new medicines can mean for patients and the people who love them. 

Today, as Senior Director, External Innovation, that experience continues to shape the way he approaches every opportunity. Beyond the science and the data, he sees the potential human impact behind every innovation Ipsen explores. 

For Jon, the value of a new medicine is measured not only by clinical outcomes, but also by the time it can give patients and families, and the moments that time can make possible. 

His story is a reminder that innovation can create something precious: more time together. Sometimes, even one extra year can make all the difference.

For Elise, illness has always been something very real and personal. Although she does not work directly with patients, she understands what it means to support a loved one through illness. It is an experience that showed her how quickly a diagnosis can impact daily life and reshape family routines. 

Between medical appointments, administrative tasks, and unexpected challenges, families often have to adapt to a new reality. Over time, Elise realized that caregiving is not only about supporting someone through treatment, but also about finding the right balance between caring for a loved one, personal life, and professional responsibilities. 

Some situations left a lasting impression on her, particularly seeing children in hospital whose parents could not always be by their side. These moments highlighted a reality many caregivers face: wanting to be present for a loved one while managing the demands of everyday life. 

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This experience reinforced her belief that presence, listening, and emotional support are just as important as practical assistance. Yet caregivers often remain behind the scenes, despite the essential role they play throughout the care journey. 

With this in mind, Ipsen has introduced caregiver leave. This initiative gives employees the time they may need to support a family member or loved one when circumstances require it, without having to choose between their caregiving responsibilities and their professional commitments. 

For Elise, this initiative is first and foremost an acknowledgment of the reality many families experience. It reflects the belief that caring for people also means supporting those who care for them every day. 

“We need to make sure that it’s scalable, safe, and reproducible—so that every patient gets the same medicine every time.”  

As a CMC lead focused on new asset development, Ian Fox works at the intersection of research and application—taking promising molecules and turning them into real-world treatments. He explains his work as “a kind of bridge between early research identification of a target area and making the end product a sustainable, controlled, safe, quality product that can meet a patient’s needs.” 

Ian supports the push from medicinal chemistry into clinical development, translating discovery into consistent, high-quality products. That means evaluating manufacturing processes early, making sure they are viable not just in theory but in scale. “It has to be always the same quality, the same dose, the same perfect medicine every time,” he says. 

Ian did not begin in development. His journey started in commercial manufacturing, then he saw an opportunity to move closer to impact. “The move into pharmaceutical development gives me the opportunity to reach more patients and change their lives.” 

That shift required growth. He “climbed the ladder” and completed a master’s focused on innovative manufacturing. Ipsen’s structure allowed him to move across teams, expand his technical scope, and step into a role where science connects directly to patients. 

He believes the future lies in better tools and deeper collaboration. “With advancements in AI, we might reduce the risk or the level of testing we have to execute—through more effective modeling,” he says. “Tools like this will aid scientists and let us focus more on the work itself.” 

In the end, for Ian, everything comes together through collaboration. “If we can’t collaborate, the product doesn’t make it to market. By working together, we work towards delivering the same results for every patient every time.”  

What a start to 2026! We announced excellent financial results today.

Total sales grew by 23.5% at constant exchange rates, driven by double-digit sales growth in all three therapeutic areas: +15.6% in Oncology, +108.0% in Rare Disease and +16.6% in Neuroscience.

This follows a series of announcements that highlight the great progress we have made since January: two acquisitions and three positive topline results in clinical trials in both chronic and episodic migraine, as well as in primary biliary cholangitis.