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About Orphalan

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.

Our Story

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.

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Orphalan team collaboration

Our Journey

  1. 2011

    Company Founded

    Orphalan established in Paris with a mission to serve rare disease patients across Europe

  2. 2017

    Cuprior® EU Approval

    Cuprior® received European Commission approval for the treatment of Wilson's disease

    2019 First country launches across Europe
  3. 2021

    Global Expansion

    Orphalan begins its international expansion beyond Europe

    United States

    2021 US subsidiary established
    2024 Cuvrior® US launch

    China

    2024 Approval received by China's National Medical Products Administration (NMPA)
    2025 Ke Pei Ou (科佩欧®) China launch
  4. 2025

    Acquisition d'Orphelia

    Orphalan acquired Orphelia, joining forces to strengthen rare disease commitment and expand capabilities

OUR VALUES

What Drives Us

Our work is shaped by our commitment to patients. Our approach is guided by five values.

Impactful

Making a meaningful difference to people living with rare diseases, who have limited or no options for treatment.

Committed

Persevering relentlessly and exercising patience to find the right solution for patients with unaddressed medical needs.

Collaborative

Connecting our expertise, patient insights and innovative thinking to work together in developing treatments to improve patients' lives.

Experts

Using the best experts, scientists and academic institutions to further pharmaceutical development, combining clinical evidence with experience.

Dynamic

Moving and adapting quickly to solve any challenge that may arise and proactively offering services and solutions for patients who need them.

Patient care and support

Our mission is simple: to ensure our therapies reach every patient, regardless of how rare their condition may be. Every patient matters.

Learn more about our patient-centric approach
NOTRE IMPACT

Orphalan by the Numbers

Our global reach and commitment to rare disease patients

Join Our Mission

Whether you are a patient, healthcare professional or potential partner, we would love to connect with you.

BACKED BY

Our Investors

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.

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Our Work With the Medical Community

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.

Investigator-Initiated Research

Supporting independent research initiatives to advance scientific understanding in rare diseases

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iWilson's Disease Registry

International patient registry advancing Wilson's disease research through real-world data

Explore Registry

Patient Support Groups

Connecting patients with support networks and resources for rare disease communities

View Resources
MLR Approval Code: ORP-EU-2026-001 • Approved: October 2026
For Healthcare Professionals and Patients in Europe and regions where Orphalan operates