Orphalan
Orphalan SA is a privately held Paris-based pharmaceutical company that develops and commercializes orphan drug treatments for rare diseases, anchored by trientine tetrahydrochloride products for Wilson's disease sold in 30+ countries and an expanding pediatric rare disease franchise acquired from Orphelia Pharma.
- Company typePrivate
- Founded2011
- HeadquartersParis, France
- Headcount—
- GTM typeB2B
- OfferingHardware or Manufacturing
What Orphalan does
Orphalan SA is a privately held French pharmaceutical company founded in 2011 and headquartered in Paris that develops and commercializes treatments for rare diseases, anchored by a franchise in Wilson's disease — a rare genetic disorder of copper metabolism. The company's core technology is a proprietary trientine tetrahydrochloride (TETA-4HCl) formulation marketed under the brand names Cuprior (EU, UK, Saudi Arabia, Switzerland, Colombia, New Zealand), Cuvrior (United States, approved by the FDA in May 2022), and Ke Pei Ou (China, NMPA-approved January 2024). The FDA approval marked the first new Wilson's disease treatment authorized in the United States in over five decades and was supported by the CHELATE Phase 3 trial, the first prospective randomized comparison of trientine tetrahydrochloride versus d-penicillamine, published in The Lancet Gastroenterology & Hepatology. Orphalan has also developed a proprietary Non-Ceruloplasmin Copper (NCC) assay in consultation with the FDA for more accurate disease monitoring, and operates the International Wilson's Disease Patient Registry (iWD Registry) as a real-world evidence platform.
The company generates revenue through direct prescription pharmaceutical product sales in markets where it operates its own commercial organization (Europe, United States) and through distribution partnerships elsewhere, notably the SPH Kyuan Trade (Shanghai Pharma Group) partnership for China. Its customer base is concentrated in specialist prescribers — hepatologists, neurologists, and pediatric specialists — treating a narrow, orphan-indication patient pool across more than 30 countries, with over 1,800 patients treated cumulatively. Following the December 2025 acquisition of Orphelia Pharma, Orphalan expanded into rare and serious pediatric diseases in neurology and oncology, including KIZFIZO, a first liquid oral formulation of temozolomide for relapsed or refractory neuroblastoma developed with Gustave Roussy. With 100+ employees, dedicated teams in 20+ countries, and a hybrid direct-sales and partner-distribution go-to-market model, Orphalan functions as a vertically integrated rare disease commercial platform rather than a single-asset biotech.
Orphalan firmographics
Firmographics- Name
- Orphalan
- Legal name
- Orphalan SA
- Website
- https://www.orphalan.com
- Company type
- Private
- Founded year
- 2011
- Operating status
- Operating
- Short description
- Orphalan SA is a privately held Paris-based pharmaceutical company that develops and commercializes orphan drug treatments for rare diseases, anchored by trientine tetrahydrochloride products for Wilson's disease sold in 30+ countries and an expanding pediatric rare disease franchise acquired from Orphelia Pharma.
- Ownership category
- akta.pro rank
Orphalan industry classification
Industry- Product category
- Orphan Pharmaceuticals
- NAICS
- Pharmaceutical and Medicine Manufacturing (3254)
- SIC
- Wholesale-Drugs, Proprietaries & Druggists' Sundries (5122)
- akta.pro primary industry
- Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)
- akta.pro secondary industries
- Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
Keywords
Where Orphalan is headquartered
LocationHeadquarters
- HQ city
- Paris
- HQ country
- France
- HQ region
- Europe
Offices11 records
Markets served
Orphalan business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Operations, Personnel, Marketing or Sales, Supply Chain, Infrastructure
Revenue model
- Pharmaceutical Product Sales: Revenue generated from sales of trientine tetrahydrochloride products (Cuprior, Cuvrior, Ke Pei Ou) for Wilson's disease treatment. Products are available in 30+ countries under various brand names.
- Drug Development and Commercialization: Orphalan identifies, develops and delivers innovative treatments for rare diseases. The company commercializes its own products through direct sales teams and distribution partnerships.
Go-to-market motion3 records
Distribution channels4 records
Marketing channels7 records
Orphalan product offering
Product offeringCore offering
Orphalan identifies, develops, and commercializes orphan drug treatments for rare diseases, with a primary focus on Wilson's disease. Its lead product is a trientine tetrahydrochloride (TETA-4HCl) formulation marketed under multiple brand names (Cuprior, Cuvrior, Ke Pei Ou) across 30+ countries. The company also develops pediatric rare-disease treatments such as KIZFIZO, a liquid oral formulation of temozolomide for relapsed or refractory neuroblastoma in children.
Differentiator
Problem solved
Functional benefit
Brands
- Cuprior: Trientine tetrahydrochloride product marketed in EU, UK, Saudi Arabia, Switzerland, Colombia, and New Zealand for Wilson's Disease treatment
- Cuvrior
- Ke Pei Ou (科佩欧)
Products and services
- Cuprior Oral trientine tetrahydrochloride formulation approved for Wilson's disease maintenance therapy in adults and children aged 5 years and older who are intolerant to penicillamine; marketed in Europe, UK, Saudi Arabia, Switzerland, Colombia, and New Zealand.
- Cuvrior Trientine tetrahydrochloride (TETA-4HCl) approved by the FDA for adult patients with stable Wilson's disease who are de-coppered and tolerant to penicillamine; the first FDA-approved Wilson's disease therapy in over 50 years.
- Ke Pei Ou (科佩欧®) Trientine tetrahydrochloride product marketed in China for Wilson's disease treatment in children aged five years and older and adults intolerant to penicillamine therapy.
- KIZFIZO Liquid oral formulation of temozolomide developed in collaboration with Gustave Roussy for treating relapsed or refractory neuroblastoma in children; first liquid oral formulation of temozolomide with orphan drug designations in Europe and the US.
Quantifiable outcome
- 50% of patients treated with trientine tetrahydrochloride achieved pre-specified composite endpoint vs 24% with d-penicillamine
- +2 more outcomes
Companies that use Orphalan
Customer profileNamed customers1 record
Segments3 records
Ideal customer profiles3 records
Orphalan technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Orphalan partnerships and signals
Strategic signalPartnerships
Seven partnerships are on record, tiered core and minor.
- Orphelia PharmacoreOrphalan acquired Orphelia Pharma, a French pharmaceutical company dedicated to rare and serious pediatric diseases in neurology and oncology. This acquisition expands Orphalan's therapeutic portfolio and strengthens its position as a European leader in rare diseases.
- MAP InternationalminorPartnership with MAP International to provide Cuvrior (trientine tetrahydrochloride) to underserved communities globally. MAP International is a Christian health organization committed to providing essential medicines to those in need.
- Wilson Disease Association (WDA)minorPartnership with Wilson Disease Association to provide Cuvrior to underserved communities. WDA is a patient advocacy organization supporting individuals and families affected by Wilson's disease.
- SPH Kyuan Trade (Kyuan Xinhai (Beijing) Medical Products Trade Co., Ltd)coreStrategic partnership with SPH Kyuan Trade, a leading pharmaceutical distributor in China and subsidiary of Shanghai Pharma Group, one of China's largest healthcare conglomerates. Partnership to transform access to Wilson's disease treatment for Chinese patients.
- International Wilson's Disease Patient Registry (iWD Registry) PartnerscoreOrphalan established the first global Wilson's Disease Patient Registry in collaboration with international physicians and research institutions. Initial enrollment sites include Children's Memorial Health Institute (Warsaw) and Institute of Psychiatry and Neurology (Warsaw), with plans to expand to Belgium, Spain, Germany, UK, and France.
- Gustave RoussycoreCollaboration with Gustave Roussy for development of KIZFIZO, the first liquid oral formulation of temozolomide for treating relapsed or refractory neuroblastoma in children. Gustave Roussy is a leading cancer research center.
- Wuhan Tongxin (Wilson Disease Patient Group in China)minorWilson disease patient group in China that expressed support for NMPA approval of Orphalan's trientine tetrahydrochloride product.
Scale indicators6 records
Recent moves6 records
Expansion highlights7 records
Orphalan competitors and assessment
Company assessmentEmerging players
- Helsinn Group: Swiss privately-held specialty pharma focused on oncology and rare diseases with global commercial partnerships. Comparable as a mid-sized European orphan-drug developer/commercializer with selective geographic expansion.
- Orphelia Pharma: French pediatric rare-disease company now wholly owned by Orphalan (acquired December 2025). Directly comparable as the developer of KIZFIZO and a peer-now-subsidiary in pediatric orphan therapeutics.
- Ultragenyx Pharmaceutical: Clinical-to-commercial rare-disease company focused on genetic and metabolic disorders. Comparable in pursuing orphan drug approvals across multiple jurisdictions for ultra-rare conditions.
Broad incumbents
- BioMarin Pharmaceutical: US rare-disease specialist focused on genetic and metabolic disorders with global commercialization. Comparable in targeting small, genetically-defined patient populations with premium-priced orphan therapies.
- Swedish Orphan Biovitrum (Sobi): European-headquartered rare-disease specialist with a portfolio spanning hematology, immunology, and specialty metabolic disorders. Highly comparable in size, geographic footprint, and rare-disease commercial approach.
- Jazz Pharmaceuticals: Specialty pharma focused on rare neurological and oncology indications with global commercial reach. Comparable in targeting small specialist prescriber bases with high-value orphan therapies.
- Genzyme (Sanofi): Pioneer in rare-disease commercialization (Cerezyme, Fabrazyme, Myozyme). Several Genzyme-trained executives now lead Orphalan, and the operational model — direct rare-disease sales infrastructure globally — closely mirrors Orphalan's.
- Vertex Pharmaceuticals: Commercial-stage rare-disease company with strong global infrastructure in genetic diseases (cystic fibrosis, sickle cell). Comparable in leveraging orphan drug exclusivity and global rollout of specialty therapeutics.
- Alexion (AstraZeneca Rare Disease): Global rare-disease leader (Soliris, Strensiq, Kanuma) operating with a similar specialty pharma commercial model targeting rare metabolic and neurological disorders. Comparable in go-to-market approach for ultra-rare conditions.
Direct peers
- Recordati Rare Diseases: Recordati's rare disease arm markets Wilzin (zinc acetate), a competing first-line Wilson's disease maintenance therapy. Most direct competitor in Orphalan's core indication, targeting the same prescribers and patient populations.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks6 records
Key highlights7 records
Customer concentration
Orphalan social profiles
Digital presenceOrphalan financial estimates
Financial estimateRevenue estimate
Valuation estimate
Orphalan leadership team
Management profileNumber of profiles
Profiles6 records
Orphalan subsidiaries and ownership
Company hierarchySubsidiaries1 record
Orphalan funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Orphalan M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Orphalan
What does Orphalan do?
Orphalan identifies, develops, and commercializes orphan drug treatments for rare diseases, with a primary focus on Wilson's disease. Its lead product is a trientine tetrahydrochloride (TETA-4HCl) formulation marketed under multiple brand names (Cuprior, Cuvrior, Ke Pei Ou) across 30+ countries. The company also develops pediatric rare-disease treatments such as KIZFIZO, a liquid oral formulation of temozolomide for relapsed or refractory neuroblastoma in children.
Is Orphalan a public or private company?
Orphalan is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was Orphalan founded?
Orphalan was founded in 2011.
Where is Orphalan based?
Orphalan is headquartered in Paris, France, in the Europe region.
How does Orphalan make money?
Two revenue lines are on record. Pharmaceutical Product Sales are the primary driver. The others are drug Development and Commercialization.
Who are Orphalan's main competitors?
Emerging players on record are Helsinn Group, Orphelia Pharma and Ultragenyx Pharmaceutical. Broad incumbents are BioMarin Pharmaceutical, Swedish Orphan Biovitrum (Sobi), Jazz Pharmaceuticals, Genzyme (Sanofi), Vertex Pharmaceuticals and Alexion (AstraZeneca Rare Disease). Recordati Rare Diseases is listed as a direct peer.
Does Orphalan have an API?
No public API is recorded for Orphalan.
What industry is Orphalan in?
Orphalan's product category is Orphan Pharmaceuticals. Its primary akta.pro industry code is HLAIAIAA, Genetic & Genomic Rare Disease Therapeutics, with a secondary code of HLAIAIAN, Rare Pediatric & Congenital Disorder Therapies. Its NAICS code is 3254 and its SIC code is 5122.