ORPHELIA Pharma
ORPHELIA Pharma is a Paris-based pharmaceutical company that develops and markets age-appropriate formulations of orphan drugs for rare pediatric diseases in neurology (Kigabeq/vigabatrin) and oncology (KIZFIZO/temozolomide), serving children with conditions such as pediatric epilepsy and high-risk neuroblastoma through academic and distribution partnerships.
- Company typePrivate
- Founded2015
- HeadquartersParis, France
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What ORPHELIA Pharma does
ORPHELIA Pharma SAS is a Paris-based pharmaceutical company that develops and commercializes age-appropriate formulations of drugs for rare pediatric diseases in neurology and oncology. Its commercial portfolio is anchored by Kigabeq (vigabatrin scored soluble tablets, 100mg and 500mg), which received EU Paediatric-Use Marketing Authorization (PUMA) in 2018 and is distributed in France, the UK, and the Baltic states via regional partners including Laboratory Biocodex and UAB ENTAFARMA. Its second lead asset, KIZFIZO (temozolomide oral suspension 40mg/ml), is a taste-masked, ready-to-use pediatric formulation for relapsed or refractory high-risk neuroblastoma that holds Orphan Drug Designation from both the EMA and FDA and was granted Early Access Authorization in France in March 2022; however, the CHMP adopted a negative opinion on its EU Marketing Authorization Application in November 2024, confirmed on 27 February 2025, and the company is pursuing alternative regulatory pathways.
The company operates a lean, partnership-driven model: it works with world-leading academic centers (Gustave Roussy since 2018, University of Birmingham, Oscar Lambret) for clinical development and with French and European industrial partners for manufacturing. Revenue mechanics combine direct pharmaceutical product sales with regional distribution agreements and licensing contracts for global reach. The company was founded by Hugues Bienaymé in 2007 (originally as TargeOn, rebranded to ORPHELIA Pharma in 2015), was backed by OCTALFA (the Alberici family holding) since 2015 alongside Pierre Fabre Médicament and three French family offices added in 2019, and was acquired by Orphalan in December 2025 to strengthen Orphalan's pediatric rare disease franchise.
ORPHELIA Pharma firmographics
Firmographics- Name
- ORPHELIA Pharma
- Legal name
- ORPHELIA Pharma SAS
- Website
- https://orphelia-pharma.eu
- Company type
- Private
- Founded year
- 2015
- Operating status
- Acquired
- Headcount range
- 1–10 employees
- Short description
- ORPHELIA Pharma is a Paris-based pharmaceutical company that develops and markets age-appropriate formulations of orphan drugs for rare pediatric diseases in neurology (Kigabeq/vigabatrin) and oncology (KIZFIZO/temozolomide), serving children with conditions such as pediatric epilepsy and high-risk neuroblastoma through academic and distribution partnerships.
- Ownership category
- akta.pro rank
ORPHELIA Pharma industry classification
Industry- Product category
- Pediatric Specialty Pharmaceuticals
- NAICS
- Pharmaceutical and Medicine Manufacturing (3254), Biological Product (except Diagnostic) Manufacturing (325414), Medical and Diagnostic Laboratories (6215)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Pediatric OTC Medicines (HLAIADAL)
- akta.pro secondary industries
- Endocrinology & Reproductive Health Pharmaceuticals (incl. Women’s Health) (HLAIAAAJ), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
Keywords
Where ORPHELIA Pharma is headquartered
LocationHeadquarters
- HQ city
- Paris
- HQ country
- France
- HQ region
- Europe
Offices2 records
Markets served
ORPHELIA Pharma business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Supply Chain, Operations, Marketing or Sales
Revenue model
- Pharmaceutical product sales: Development, manufacturing, and commercialization of orphan and pediatric medicines. Revenue generated from approved drugs and products under regulatory review.
- Distribution agreements: Regional distribution agreements in European territories through partners like Laboratory Biocodex and UAB ENTAFARMA. Licensing contracts to ensure global availability of products.
Go-to-market motion2 records
Distribution channels4 records
Marketing channels4 records
ORPHELIA Pharma product offering
Product offeringCore offering
ORPHELIA Pharma develops, manufactures, and commercializes age-appropriate pediatric pharmaceutical formulations for the treatment of rare diseases in neurology and oncology. Its commercial product is Kigabeq® (vigabatrin scored soluble tablets) granted EU paediatric-use marketing authorization, while Kizfizo® (temozolomide oral suspension) is being pursued for pediatric relapsed or refractory high-risk neuroblastoma. The company also runs an undisclosed pediatric controlled-release orphan neurology project.
Product overview
ORPHELIA Pharma is a French pharmaceutical company that develops and markets medicines for the treatment of rare pediatric diseases in two main therapeutic areas: neurology and oncology. The company operates a portfolio model with Kigabeq® (an approved EU product for pediatric epilepsy) and Kizfizo® (an oncology product under regulatory review for pediatric neuroblastoma), plus an undisclosed neurology project in development. The company was acquired by Orphalan in December 2025. Products are distributed through partnerships including Laboratoire Biocodex for Kigabeq® in France and UK, and UAB ENTAFARMA for Baltic countries.
Differentiator
Problem solved
Functional benefit
Products and services
- Kigabeq® Paediatric-use marketing authorized (PUMA) product consisting of vigabatrin scored soluble tablets in 100 mg and 500 mg strengths, indicated for the treatment of pediatric encephalopathic epilepsy. The age-appropriate, breakable soluble formulation fully responds to EMA recommendations for pediatric medicinal products.
- Kizfizo® Temozolomide oral suspension (40 mg/ml) developed as a ready-to-use, taste-masked liquid pediatric formulation of temozolomide for relapsed or refractory high-risk neuroblastoma. Supplied as a kit containing 1 bottle filled with 18 ml of suspension and 1 dosing oral syringe. Delivers accurate dose in small volume while avoiding drug handling and caregiver exposure. Holds Orphan Drug Designation from EMA and FDA.
- Undisclosed pediatric orphan neurology project New orphan drug in development for the treatment of severe and rare pediatric encephalopathic epilepsy in patients from age 1 year, using a specific pediatric controlled-release formulation.
Quantifiable outcome
- Kigabeq received EU marketing authorization in 2018 and is available in several countries
- +2 more outcomes
Companies that use ORPHELIA Pharma
Customer profileNamed customers3 records
Segments3 records
Ideal customer profiles3 records
ORPHELIA Pharma technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
ORPHELIA Pharma partnerships and signals
Strategic signalPartnerships
Nine partnerships are on record, tiered minor, core and secondary.
- Jones DayminorLegal advisors to Orphelia Pharma in the acquisition by Orphalan in December 2025.
- Oscar Lambret clinical centercoreCollaboration to study KIZFIZO in the MetroWILMS-1906 clinical trial (EudraCT: 2021-002540-67) for treatment of refractory or relapsed nephroblastoma (Wilms tumor) in pediatric patients.
- University of BirminghamcoreCollaboration to leverage patient-level data from the BEACON refractory or relapsed neuroblastoma trial to support marketing authorization of KIZFIZO. The University of Birmingham Enterprise manages the data sharing agreement. Orphelia Pharma also supports the BEACON-2 follow-on trial.
- Gustave RoussycoreGustave Roussy, one of the largest cancer centers in Europe, has collaborated with ORPHELIA Pharma since 2018 on developing KIZFIZO (temozolomide oral suspension). The formulation was developed through collaboration between pharmacists and clinicians at Gustave Roussy hospital and Orphelia Pharma's development team.
- Laboratory BiocodexcoreDistribution partner for Kigabeq in France and United Kingdom. Laboratory Biocodex, based in Gentilly, France, handles distribution of the vigabatrin pediatric formulation.
- UAB ENTAFARMAsecondaryDistribution partner for Kigabeq in Baltic countries (Širvintai district municipality, Lithuania).
- Academic PartnerscoreORPHELIA Pharma conducts research projects through academic partnerships to identify key medical needs and assess potential drug candidates. World-leading academic clinical and research teams provide specialized expertise.
- Industrial PartnerscoreIndustrial partnerships essential for manufacturing prototypes, pilot batches, clinical batches, and commercial batches. ORPHELIA Pharma works mainly with French and European industrial partners.
- SIOPEN and ITCCsecondaryEuropean expert groups involved in the BEACON trial: SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer) collaborated with University of Birmingham and Orphelia Pharma.
Scale indicators4 records
Recent moves6 records
Expansion highlights5 records
ORPHELIA Pharma competitors and assessment
Company assessmentBroad incumbents
- Orphalan: Parent company that acquired ORPHELIA in December 2025; develops and commercializes orphan drugs including Cuprior for Wilson's disease, with presence in 38 countries. Most directly comparable as a rare-disease specialty pharma now absorbing ORPHELIA's pediatric portfolio.
- Swedish Orphan Biovitrum (Sobi): Swedish rare-disease leader with a strong pediatric portfolio across hematology, immunology, and specialty care. Comparable as an established rare-disease commercial platform with overlap in pediatric orphan therapeutics.
- Alexion (AstraZeneca Rare Disease): Global rare-disease franchise within AstraZeneca developing and commercializing therapies for ultra-rare conditions, including pediatric patients. Comparable as a large incumbent in the orphan-drug commercial space.
- BioMarin Pharmaceutical: US rare-disease biotech with multiple approved orphan drugs, including pediatric indications (e.g., enzyme replacement therapies). Comparable for rare pediatric disease commercial and regulatory playbook.
- Ultragenyx Pharmaceutical: Commercial-stage rare-disease company developing therapies for serious and ultra-rare genetic diseases, many of them pediatric. Comparable orphan-drug development model with multiple regulatory designations.
Direct peers
- Recordati Rare Diseases (formerly Orphan Europe): European rare-disease specialist with a portfolio that historically included pediatric orphan drugs. Highly comparable business model: developing and commercializing niche therapeutics for small patient populations, including pediatric indications.
- Proveca Pharma: European pharma focused exclusively on developing age-appropriate medicines for children, including formulations for neurological and rare pediatric conditions. Closest direct competitor in the niche of pediatric-specific drug reformulation.
- Ethypharm: French specialty pharmaceutical company with deep expertise in pediatric and CNS formulations, including liquid and oral dose forms. Comparable for proprietary formulation know-how targeting pediatric and CNS indications.
Others
- Pierre Fabre Médicament: Long-term shareholder of ORPHELIA since founding and a French pharmaceutical group with oncology and rare-disease exposure. Relevant as historical investor and a European pharma with adjacent therapeutic focus.
Regional players
- Laboratoire Biocodex: French family-owned pharma that distributes Kigabeq in France and the UK for ORPHELIA. Comparable as a European specialty pharma with pediatric-relevant product lines and a similar privately-held, family-controlled profile.
Market position
Strengths4 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
ORPHELIA Pharma social profiles
Digital presenceORPHELIA Pharma financial estimates
Financial estimateRevenue estimate
Valuation estimate
ORPHELIA Pharma leadership team
Management profileNumber of profiles
Profiles1 record
ORPHELIA Pharma funding detail
Funding detailFunding overview
Funding rounds2 records
Investors1 record
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
ORPHELIA Pharma M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about ORPHELIA Pharma
What does ORPHELIA Pharma do?
ORPHELIA Pharma develops, manufactures, and commercializes age-appropriate pediatric pharmaceutical formulations for the treatment of rare diseases in neurology and oncology. Its commercial product is Kigabeq® (vigabatrin scored soluble tablets) granted EU paediatric-use marketing authorization, while Kizfizo® (temozolomide oral suspension) is being pursued for pediatric relapsed or refractory high-risk neuroblastoma. The company also runs an undisclosed pediatric controlled-release orphan neurology project.
Is ORPHELIA Pharma a public or private company?
ORPHELIA Pharma is a private company. It is classified as corporate owned and is currently acquired.
When was ORPHELIA Pharma founded?
ORPHELIA Pharma was founded in 2015. It employs 1 to 10 people.
Where is ORPHELIA Pharma based?
ORPHELIA Pharma is headquartered in Paris, France, in the Europe region.
How does ORPHELIA Pharma make money?
Two revenue lines are on record. Pharmaceutical product sales are the primary driver. The others are distribution agreements.
Who are ORPHELIA Pharma's main competitors?
Broad incumbents on record are Orphalan, Swedish Orphan Biovitrum (Sobi), Alexion (AstraZeneca Rare Disease), BioMarin Pharmaceutical and Ultragenyx Pharmaceutical. Direct peers are Recordati Rare Diseases (formerly Orphan Europe), Proveca Pharma and Ethypharm. Pierre Fabre Médicament is listed as an others. Laboratoire Biocodex is listed as a regional player.
Does ORPHELIA Pharma have an API?
No public API is recorded for ORPHELIA Pharma.
What industry is ORPHELIA Pharma in?
ORPHELIA Pharma's product category is Pediatric Specialty Pharmaceuticals. Its primary akta.pro industry code is HLAIADAL, Pediatric OTC Medicines, with a secondary code of HLAIAAAJ, Endocrinology & Reproductive Health Pharmaceuticals (incl. Women’s Health). Its NAICS code is 3254 and its SIC code is 2836.