This strategic transaction further strengthens Orphalan’s position as a European leader with a global commercial footprint in rare diseases and expands our therapeutic portfolio.
Paris, December 1, 2025 – Orphalan, a global pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a French pharmaceutical company dedicated to rare and serious pediatric diseases, particularly in neurology and oncology.
Key highlights of the acquisition include:
- Expansion of our capabilities in rare neurological and oncological pediatric diseases
- Greater reach and improved access to medicines for children and adults with rare conditions
- A unique platform combining scientific excellence, agility, and global commercialization
By combining our complementary expertise, we are poised to accelerate innovation and improve access to life-changing treatments for patients worldwide. As we move forward together, our teams remain dedicated to ensuring continuity of care and ongoing support for our partners and patients.
Executive Statements:
“The acquisition of Orphelia Pharma reinforces our mission to bring innovative and accessible treatments to patients with rare diseases wherever they live,” said Dr. Naseem Amin, Chief Executive Officer of Orphalan. “Together, we are building a unique platform that combines scientific excellence, agility, global development and commercial capabilities to advance care for patients of all age groups with orphan disorders.”
“At Orphelia Pharma, we have devoted ourselves to developing safe and effective pediatric medicines that address major unmet needs in rare diseases,” added Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma. “Joining forces with Orphalan allows us to expand the reach and impact of our medicines by facilitating and accelerating access to children who need them most.”
Gilles Alberici, Executive Chairman and Chief Executive Officer of Orphelia Pharma, commented: “Bringing these complementary companies together marks a significant milestone for both our organizations and for the broader rare-disease patient population. As a long-term lead investor through OCTALFA, I am proud of the journey we have made and excited about the next steps of the combined company, “
About Orphalan
Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Born of a successful collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson’s disease, now licensed and authorized for use in 38 countries and currently marketed in 28 countries. Orphalan’s ambition is to reduce diagnostic delays and make innovative orphan medicines accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals.
About Orphelia Pharma
Orphelia Pharma is a French pharmaceutical company based in Paris and Lyon, focused on the development and commercialization of medicines for rare and serious pediatric diseases. With one approved drug in the European Union (Kigabeq®) and another, in collaboration with Gustave Roussy (Villejuif, France) soon to be submitted for regulatory review in the EU (Kizfizo®), Orphelia Pharma has established regional distribution agreements in Europe and other territories and collaborates with academic and industrial partners to advance research in orphan diseases.
GL-ORPH-CORP-25029 – November 2025
