Orphalan announces acquisition of Orphelia Pharma

PARIS, Dec. 12, 2025 /PRNewswire/ -- Orphalan, an international pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a company committed to treating rare and severe pediatric diseases, notably in neurology and oncology.

This strategic transaction strengthens Orphalan's position as a European leader with a global commercial footprint in rare diseases, expanding its therapeutic portfolio.

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 bringing together complementary expertise, the companies are now positioned to accelerate innovation and improve access to transformative treatments for patients around the world. As they enter this new stage, their teams remain fully committed to ensuring continuity of care and supporting their partners and patients.

Dr. Naseem Amin, Chief Executive Officer of Orphalan said:

"The acquisition of Orphelia Pharma reinforces our mission: to provide innovative and accessible treatments to patients living with rare diseases, wherever they are.  

"Together, we are building a unique platform that brings together scientific excellence, agility, international development and commercial capabilities to advance care for patients of all ages suffering from orphan diseases."

Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma, added:

"At Orphelia Pharma, we have dedicated ourselves to developing safe and effective pediatric medicines that address major unmet medical needs in rare diseases.

"Joining Orphalan will allow us to increase the impact of our treatments and make them available more rapidly to the children who need them most."

Gilles Alberici, Chief Executive Officer of Orphelia Pharma, commented:

"Bringing together our two complementary companies marks an important milestone, both for our organizations and for the broader community of patients living with rare diseases."

About Orphalan

Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Originating from a collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson's disease, now authorized in 38 countries and marketed in 28.

Orphalan's ambition is to reduce diagnostic delays and make innovative orphan drugs accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals.

Orphalan's shareholders include Bpifrance, Advent, and the management team.

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon, specializing in the development and commercialization of medicines for rare and severe pediatric diseases. With one authorized medicine in the European Union (Kigabeq®) and a second under regulatory review (Kizfizo®), Orphelia Pharma has established regional distribution agreements across Europe and other regions and collaborates with academic and industrial partners to advance research in rare diseases.

Further information:

Bien Commun Advisory 
M. Benedict Brodrick
+33.6.76.14.02.02
b.brodrick@bcadvisory.fr

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