Orphalan

Orphalan driving action for rare diseases   Orphalan was proud to actively contribute to the event "Accelerating innovation for Rare Diseases: from research to patient access, partnerships & perspectives," co-organized by France Biotech and Institut Imagine in Paris.   One of the key highlights of the day was the presentation of the White Paper from France Biotech’s Rare Disease Expert Group, co-led by our France Managing Director, Nolwenn Le Rhun.   What’s in our white paper? Bridging the funding gap: Recommendations for sustainable financing models to support rare disease R&D from early-stage research to commercialization Market access & pricing: Proposals for new evaluation methods tailored to rare diseases, ensuring that innovative treatments reach patients faster. Harnessing real-world data & AI: Promoting advanced data-driven approaches to improve drug assessment and reimbursement processes. Stronger public-private partnerships: Encouraging collaboration between biotech innovators, research institutions, and policymakers to drive impact.   🚀 Key Moment: Nolwenn Le Rhun’s roundtable on market Access & data-Driven evaluation Nolwenn moderated an insightful discussion on how data can be better leveraged to support regulatory decisions and improve patient access. The panel brought together key industry and regulatory leaders to address: 🔹 The role of real-world data in complementing clinical trials, particularly when patient populations are small. 🔹 Challenges in regulatory acceptance of AI-driven insights and real-world evidence. 🔹 The need for adaptive pricing models to ensure rare disease treatments remain accessible and financially viable. 🔹 The importance of patient advocacy groups in shaping the future of rare disease evaluation.   At Orphalan, we are fully committed to translating innovation into real impact for patients. This event was a key step in pushing for stronger policies and faster access to life-changing treatments.   Thank you to all the experts, partners, and advocates who contributed to these vital discussions! Nolwenn Le Rhun Magali Fournier Pacanowski Antoine Ferry Constance Sabbagh Solange Roumengous Laure Jamot Mathieu Bouthemy Sophie Pelé Geraldine Van den Broek Fanny ROTH Hélène Chautard Bien Commun Advisory Mathilde Boizon Marie-Charlotte POTET Isabelle Berard Côme de Sauvebeuf Link to the white paper in comments :

A historic milestone for Orphalan: bringing Ke Pei Ou (科佩欧®) to China!   We are proud to announce that Orphalan has become the first European pharmaceutical company to enter China’s Wilson Disease market with the launch of Ke Pei Ou (科佩欧®), our trientine tetrahydrochloride treatment. This marks a transformational moment for patients in China who previously had limited treatment options.  Dr. Naseem Amin, our CEO puts it best: "This is more than market expansion, this is bringing an important new, previously unavailable therapeutic option for rare disease patients in China. We are not just bringing a treatment to market; we are providing access to care for those who need it most."

Strengthening Our Commitment to Rare Disease Communities in China   We are thrilled to share our involvement in the recent China Rare Disease Day! On March 1st, the China TongXin Wilson Disease (WD) patient advocacy group organized an impactful rare disease education event in Wuhan.   This gathering brought together nearly 50 rare disease patients, along with pediatricians and neurologists from Wuhan, fostering valuable discussions on patient care and medical advancements. Orphalan China was honored to support this initiative which marks an important step in our collaboration with China’s Wilson Disease patient action group and the broader rare disease community. We look forward to strengthening these partnerships and continuing our mission to support patients worldwide.   #Orphalan #RareDiseaseDay #WilsonDisease #PatientAdvocacy #China #Healthcare

Reflections from our CEO, Naseem Amin, on Rare Disease Day: Today, February 28, 2025, marks Rare Disease Day, an international event that highlights the challenges faced by over 300 million people worldwide living with rare conditions.   This day is crucial for raising awareness, advocating for equitable access to diagnosis and treatment, and fostering solidarity among patients, families, and healthcare professionals. Our work at Orphalan directly contributes to advancing research and treatment for rare diseases.   At Orphalan, we are proud of the work we do every day. Together, we are making a difference.

Friday, February 28th, is #RareDiseaseDay, an important reminder to recognise the incredible strength of people and families living with a rare condition. These conditions impact over 300 million people around the world, with each patient navigating their own journey filled with obstacles, milestones, and moments of triumph. Unfortunately, many continue to struggle with access to the care, treatments, and support they desperately need. Together, by joining forces with patients, advocates, researchers, and healthcare providers, we can bring about meaningful change for those affected by these conditions. At Orphalan we work with partners to develop and deliver innovative treatments for patients living with rare diseases. Our first commercialised product is for Wilson disease, a rare genetic condition affecting 1 in 30,000 people globally. On this Rare Disease Day, we, at Orphalan, are more committed than ever to raising awareness of rare diseases globally, and we are proud to participate in the 4TH Rare Diseases Run 2025 - RUN FOR RARE to amplify our message - https://lnkd.in/dunWntG2 We invite you to watch a brief video where our Orphalan employees Share Their Colours and insights about Wilson’s Disease.   For more information about Orphalan and our goal of transforming treatments for rare diseases, please follow this link www.orphalan.com #RareDiseaseDay #ShareYourColours #RunForRare #RareDisease #OrphanDiseases

Driving innovation for rare diseases in France! At Orphalan, we are dedicated to transforming the landscape of rare disease treatments by ensuring that innovative therapies reach patients faster. But to achieve this, we must address critical barriers in funding, development, and market access.   For the past year, France Biotech’s Rare Disease Task Force, co-led by Nolwenn Le Rhun, our Managing Director in France as gathered over 50 experts from across the healthcare ecosystem—startups, researchers, clinicians, investors, policymakers, and patient associations.   Today, we are proud to unveil its white paper, a strategic roadmap to accelerate innovation and improve patient access to life-changing treatments.   Key findings & recommendations: ✅ Strengthening funding mechanisms to support innovation throughout the entire development cycle. ✅ Facilitating market access by adapting pricing frameworks and implementing performance-based agreements. ✅ Boosting investment in rare disease research to bridge the gap between scientific breakthroughs and patient solutions. ✅ Improving screening and diagnosis to ensure early and effective treatment pathways.   This White Paper is a call to action. France has the potential to be a leader in rare disease innovation. At Orphalan, we are proud to contribute to this collective effort, reinforcing our mission to develop and deliver innovative treatments for patients living with rare diseases. 

It’s the 1st International Wilson Disease Day! The Wilson Disease International Community selected 6th December as it’s the birthday of Dr. Samuel Alexander Kinnier Wilson, to highlight how important his discovery was for patients living with Wilson disease. Dr. Wilson first described this rare genetic disorder in 1912 and his knowledge has paved the way for further research, allowing patients to be diagnosed correctly and ensure their lifelong condition is manageable. At Orphalan, we are proud to support this initiative and the incredible work of patient groups and healthcare professionals worldwide. So today, we say, ‘leave no one behind’! #Wilsondisease #Raregeneticdiseases #Rarediseases #Wilsondiseaseawareness #WilsonDiseaseAwarenessDay #WilsonDiseaseAssociation #Orphalan November 2024. GL-ORPH-CORP-24007.

At Orphalan, we are committed to raising awareness and supporting the Wilson disease community. Wilson disease is a rare genetic disorder that affects about 1 in 30 000 people worldwide.¹ Symptoms only appear after copper builds up and damages organs like the liver, brain and eyes.² Early diagnosis and treatment can significantly impact how people lead their lives. Watch Cory’s journey on the Medical Stories segment airing on Public Television Stations (PBS) to discover what it’s like living with Wilson disease. https://lnkd.in/dUjmby9W #MedicalStories #Publictelevision #Wilsondisease #Rarediseases #Raregeneticdiseases #Wilsondiseaseawareness #WilsonDiseaseAwarenessDay #WilsonDiseaseAssociation #Orphalan 1. European Association for the Study of the Liver. EASL Clinical Practice Guidelines: Wilson’s disease. J Hepatol 2012;56:671–685. 2. British Liver Trust. https://lnkd.in/dJamh5hS [Accessed November 2024] November 2024. GL-ORPH-CORP-24007.

Genetic diseases can have devastating effects on patients and their loved ones. Wilson disease is a rare genetic disorder that can cause life-threatening copper (Cu) accumulation if left untreated.¹ At Orphalan, we are driven by a desire to improve the lives of people suffering with these conditions. Our goal is to make Wilson disease more manageable in the long term. Watch our video below to see how we’re helping the Wilson disease community. We invest, we care, we Cu! #Rarediseases #Raregeneticdiseases #Wilsondisease #Wilsondiseaseawareness #WilsonDiseaseAwarenessDay #WilsonDiseaseAssociation #WeCu #Orphalan 1. European Association for the Study of the Liver. EASL Clinical Practice Guidelines: Wilson’s disease. J Hepatol 2012;56:671–685. November 2024. GL-ORPH-CORP-24006.

Situation Vacant https://lnkd.in/dvEhg_PJ