Company Founded
Orphalan established in Paris with a mission to serve rare disease patients across Europe
We are an international orphan drug development and commercialisation company headquartered in Paris.
Founded in 2011, we develop and deliver innovative therapies for people living with rare and debilitating diseases.
Orphalan was established in 2011 with a clear focus: to develop and commercialise treatments for patients with rare diseases. What began as a dedicated effort in Wilson's disease has evolved into a comprehensive rare disease platform serving patients across multiple therapeutic areas.
Our primary focus is on rare, often serious diseases where current therapeutic options do not exist, are limited, or are associated with significant challenges for patients. Our efforts have resulted in the development and commercialisation of therapies in global markets, including Europe and the United States, with further submissions in progress to make our products available to patients worldwide.
In 2025, we acquired Orphelia, strengthening our capabilities and expanding our portfolio while maintaining our patient-focused approach and rare disease expertise.
En savoir plus
Orphalan established in Paris with a mission to serve rare disease patients across Europe
Cuprior® received European Commission approval for the treatment of Wilson's disease
Orphalan begins its international expansion beyond Europe
United States
China
Orphalan acquired Orphelia, joining forces to strengthen rare disease commitment and expand capabilities
Our work is shaped by our commitment to patients. Our approach is guided by five values.
Making a meaningful difference to people living with rare diseases, who have limited or no options for treatment.
Persevering relentlessly and exercising patience to find the right solution for patients with unaddressed medical needs.
Connecting our expertise, patient insights and innovative thinking to work together in developing treatments to improve patients' lives.
Using the best experts, scientists and academic institutions to further pharmaceutical development, combining clinical evidence with experience.
Moving and adapting quickly to solve any challenge that may arise and proactively offering services and solutions for patients who need them.
Our global reach and commitment to rare disease patients
Whether you are a patient, healthcare professional or potential partner, we would love to connect with you.
We are proud to be backed by a high-quality group of leading investors whose shared vision is to create a global business that delivers a meaningful and impactful difference to patients living with rare diseases.
At Orphalan, we continuously engage with healthcare professionals and patient advocacy groups in our common goal of improving care and services for patients with rare disease. Together, by taking a patient-centric approach, in partnership with healthcare professionals, our mission is to collaborate to advance knowledge in rare disease and in particular to identify and find innovative solutions for the unmet needs of patients.
Orphalan along with our network of Key Opinion Leaders strive to design meaningful and important studies to improve patient care and outcomes. It is through close collaborations with healthcare professionals and scientists, that Orphalan is able to address these unmet needs of patients with rare disease and potentially transform their lives through discovery and innovation.
Supporting independent research initiatives to advance scientific understanding in rare diseases
Learn MoreInternational patient registry advancing Wilson's disease research through real-world data
Explore RegistryConnecting patients with support networks and resources for rare disease communities
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