We Care
Patient needs inform our research and development priorities.
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Advancing Rare Disease Solutions
Rewriting the Rules in Rare Disease
Orphalan is committed to supplying therapies for rare diseases across Europe and international markets, expanding our capabilities following the acquisition of Orphelia.
- EMA Approved
- Global Compliance
- Orphelia Acquisition
1000s
Patients Helped Worldwide
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Orphalan by Numbers
Driven by science, guided by patients
Facing a rare disease can be an isolating experience for patients and their families. Orphalan aims to provide support and resources throughout their journey.
Learn about patient supportPillars of Excellence
Five core principles guide our approach, reflecting our focus on the patients we serve.
We Innovate
Researching new approaches to rare disease treatment.
Discover moreWe Solve
Focused on unmet needs in rare diseases.
Discover moreWe Reach
Expanding access to treatments across the regions we serve.
Discover moreWe Unite
One team, working toward a shared mission in rare disease.
Discover moreConditions We Focus On
Rare diseases that are underserved and require specialised expertise to treat
Wilson's Disease
A rare inherited disorder causing copper to accumulate in vital organs. Without treatment, it can cause serious liver and neurological damage. Treatment aims to help manage the condition.
Learn moreWest's syndrome
A severe form of epilepsy that begins in infancy, characterised by infantile spasms and developmental delay. Early treatment may help support better outcomes.
Learn moreGlioblastoma
A fast-growing type of brain cancer, and one of the more common types of brain tumour diagnosed in adults. Orphalan is researching new approaches to this condition.
Learn moreOur Therapeutic Portfolio
Orphalan's approved therapies for rare diseases
Cuprior® (EU)
Wilson's Disease
Also marketed as Cuvrior® in the US and Ku Pei Ou® in China
View Product InfoKigabeq® (EU)
West's Syndrome
View Product Info
PATIENT-CENTRIC APPROACH
Learn About Our Patient Commitment
It Starts With the Patient
Every day, we are driven by the stories of patients and families affected by rare diseases. Their courage inspires our research, their needs guide our innovation and their experiences inform our commitment.
Through our acquisition of Orphelia, we have strengthened our capabilities to deliver accessible, innovative therapies to those who need them most across Europe and our global markets.
- Patient support groups and advocacy partnerships
- Disease information and awareness resources
- Collaborative partnerships with patient advocacy groups
Get in touch
Reach our team with any questions
Newly diagnosed? Start here
A short FAQ for patients and families
Advocacy partners
Organisations we work with
Trusted by Healthcare Professionals Worldwide
Our commitment to quality, compliance and patient safety
European Medicines Agency (EMA) approved treatments
15+ years of dedicated rare disease expertise
Global regulatory compliance standards
Expanded capabilities through Orphelia acquisition
News & Insights
Stay informed about our latest developments, partnerships and contributions to the rare disease community
Orphalan announces European Medicines Agency (EMA) acceptance for review of Marketing Authorization Application (MAA) for KIZFIZO®
Marketing Authorization Application for KIZFIZO® accepted for review by the EMA for the treatment of malignant glioma.
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ABOUT US
Learn More About Orphalan
Connect with our team or learn more about our work in rare diseases.
MLR Approval Code: ORP-EU-2026-001
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Approved: October 2026
For Healthcare Professionals and Patients in Europe and regions where Orphalan operates