Metabolic Disorders
Rare genetic disorders affecting metabolism, including Wilson's disease
Learn MoreTherapeutic Focus Areas
Dedicated expertise in rare disease treatment
Orphalan focuses on three therapeutic areas in rare diseases: metabolic disorders (including Wilson's disease), West's syndrome and glioblastoma.
Our Areas of Expertise
We concentrate our efforts on rare diseases where specialised knowledge and dedicated research can improve patient outcomes and quality of life.
Glioblastome
A fast-growing type of brain cancer and the most common form of aggressive brain cancer in adults.
Learn MoreOur Scientific Approach
Patient-Centred Research
We design our research programmes with patients at the centre, focusing on outcomes that matter most to those living with rare diseases and their families.
Scientific Excellence
Our research is guided by rigorous scientific standards and conducted in collaboration with leading experts in each therapeutic area.
Regulatory Expertise
We work closely with regulatory authorities across multiple regions to ensure our therapies meet the highest standards for safety and efficacy.
Long-Term Commitment
Our commitment extends beyond initial approval, with ongoing research to better understand long-term outcomes and optimise treatment strategies.
Our Products
This section lists Orphalan's approved therapies by therapeutic area.