What Gen.Orph does

Gen.Orph is a privately held French pharmaceutical laboratory headquartered in Saint-Cloud, France, focused on developing and commercializing orphan drugs for rare diseases, injectable formulations for neonatal intensive care, and established medications with renewed therapeutic potential. The company was founded in 2017 by Dr. Etienne Jacob, a physician trained at Pitié-Salpêtrière and Harvard Business School, together with co-founder Patrice Layrac. Its product portfolio centers on two EMA centrally registered orphan drugs — Miglustat GEN-ORPH (oral treatment for adult type 1 Gaucher disease and for progressive neurological manifestations of Niemann-Pick Type C disease) and Ibuprofène GEN-ORPH 5 mg/ml injectable solution (for hemodynamically significant patent ductus arteriosus in premature newborns under 34 weeks gestational age, registered March 2024 and launching in 2024) — alongside two classic medications, Agyrax (meclozine for vertigo and motion sickness) and Navidoxine, which are commercialized in select international markets.

The company operates a sales-led go-to-market model, distributing products through pharmaceutical wholesale networks across Europe (France, Sweden, Norway, Denmark, Italy, Bulgaria, Germany, Portugal, Spain, UK, Belgium, Luxembourg, Hungary), the Middle East (Saudi Arabia, Oman), West Africa (Senegal, Côte d'Ivoire), and Oceania (New Zealand), with planned expansion into Mexico, Ecuador, Libya, and the Czech Republic. Miglustat is currently the most widely distributed product, available in 15 countries. Revenue is generated through one-time sales of patented and commercialized pharmaceutical products across rare disease, neonatal care, and general therapeutic segments, with pricing managed through standard pharmaceutical distribution channels and country-level regulatory pricing bodies.

Gen.Orph is a small, operationally focused organization (1-10 employees) with a dedicated leadership team covering commercial, regulatory, industrial, and financial functions, plus responsible pharmacists. The company's stated mission is to make "useful and affordable" medications accessible to patients and the international medical community, positioning itself as a value-oriented alternative in rare disease segments where therapeutic options are limited. Beyond its four commercialized products, the company indicates a pipeline of additional products in development, signaling an intent to scale its orphan drug portfolio over time.

Name

Gen.Orph

Legal name

Gen.Orph

Website

https://gen-orph.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

Gen.Orph is a French specialty pharmaceutical laboratory that develops and commercializes orphan drugs and established medications across Europe, the Middle East, Africa, and Latin America, serving patients with rare metabolic diseases, premature infants, and those with vertigo conditions.

Ownership category

akta.pro rank

Product category

Orphan Drugs / Rare Disease Pharmaceuticals

NAICS

Pharmaceutical Preparation Manufacturing (325412), Pharmaceutical and Medicine Manufacturing (3254)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industries

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Endocrinology & Metabolic Specialty Pharmaceuticals (HLAIACAD)

Keywords

• Orphan drugs
• Rare disease treatments
• Pharmaceutical manufacturing
• Neonatal care drugs
• Injectable formulations

Headquarters

HQ city

Saint-cloud

HQ country

France

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Supply Chain, Technology or R&D, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Pharmaceutical Product Sales: Gen.Orph generates revenue through the sale of patented and commercialized pharmaceutical products, including orphan drugs (Miglustat), injectable formulations (Ibuprofène), and established medications (Agyrax, Navidoxine), distributed across European and international markets. The company operates across rare disease, neonatal care, and general therapeutic segments.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels2 records

Core offering

Gen.Orph develops, registers, and commercializes pharmaceutical products focused on orphan drugs for rare metabolic and genetic diseases, injectable formulations for neonatal care, and established medications for vertigo and motion sickness. The company holds centralized European (EMA) registrations for Miglustat GEN-ORPH and Ibuprofène GEN-ORPH, and distributes its products across approximately 15 countries in Europe, the Middle East, Africa, Latin America, and Oceania through pharmaceutical wholesale networks.

Product overview

Gen.Orph is a pharmaceutical company offering a portfolio of orphan drugs for rare diseases and classic medications. The core products include Miglustat GEN-ORPH for Gaucher disease and Niemann-Pick Type C, Ibuprofène GEN-ORPH for patent ductus arteriosus in premature newborns, and commercialized products Agyrax (for vertigo and motion sickness) and Navidoxine. The company focuses on making useful and affordable medications accessible to international patients and medical professionals for rare disease treatment, classic drugs with medical and economic potential, and innovative medications for common diseases.

Differentiator

Problem solved

Functional benefit

Brands

• Miglustat GEN-ORPH: Miglustat oral treatment for adult patients with mild to moderate type 1 Gaucher disease and for progressive neurological manifestations in adult and children with Niemann-Pick Type C disease.
• Ibuprofène GEN-ORPH
• Agyrax
• Navidoxine

Products and services

• Miglustat GEN-ORPH Oral treatment for adult patients with mild to moderate type 1 Gaucher disease when enzyme replacement therapy is unsuitable, and for progressive neurological manifestations in adult and pediatric Niemann-Pick Type C patients. Centralized European registration. For metabolic disease specialists, neurologists, and hospital pharmacies.
• Ibuprofène GEN-ORPH 5 mg/ml Injectable Solution 5 mg/ml injectable solution for treatment of hemodynamically significant patent ductus arteriosus in premature newborns under 34 weeks gestational age. For neonatologists, NICU pharmacists, and hospital procurement teams.
• Agyrax (Dichlohydrate de meclozine) Meclozine dihydrochloride product for symptomatic treatment of vertigo attacks and prevention and treatment of motion sickness. For physicians, pharmacists, and patients in indicated markets.
• Navidoxine (Dichlohydrate de meclozine) Pharmaceutical product commercialized in France and Malta for its indicated use (prevention and treatment of motion sickness). For physicians, pharmacists, and patients in France and Malta.

Segments2 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Scale indicators3 records

Recent moves5 records

Expansion highlights4 records

Broad incumbents

• BioMarin Pharmaceutical: Established rare-disease pharma focused on genetic and metabolic disorders with a portfolio of EMA-approved orphan drugs. Comparable therapeutic-area focus (rare genetic/metabolic) and regulatory pathway approach, though at meaningfully greater scale and pipeline depth than Gen.Orph.
• Alexion (AstraZeneca Rare Disease): Global rare-disease leader (now part of AstraZeneca) with assets in metabolic and genetic rare diseases. Comparable to Gen.Orph in targeting rare-disease patient populations with high-priced, low-volume therapeutics, though at vastly larger scale and broader indication coverage.
• Genzyme (Sanofi): Sanofi's rare-disease division and the dominant incumbent in Gaucher disease (Cerezyme, Cerdelga/eliglustat). Directly competes with Gen.Orph's Miglustat in the Gaucher/NPC space, setting the standard of care against which Gen.Orph positions as an oral alternative for ERT-ineligible patients.
• Takeda (Rare Diseases / Shire integration): Global rare-disease franchise (built via Shire acquisition) with Vpriv and Replagal for Gaucher disease, directly competing with Gen.Orph's Miglustat in the same indication. Comparable therapeutic footprint in lysosomal storage disorders and rare metabolic diseases.

Direct peers

• Ultragenyx Pharmaceutical: Specialty rare-disease company developing and commercializing therapies for genetic and metabolic disorders (including NPC). Comparable to Gen.Orph in target disease categories (rare metabolic/genetic), EMA-oriented development, and focus on small patient populations with high unmet need.
• Orphelia Pharma: Paris-based specialty pharma focused on pediatric and rare-disease medicines, including hospital-based formulations. Closely comparable to Gen.Orph in French specialty-pharma origin, EMA-registered orphan and pediatric products, and a portfolio model of niche commercialized assets plus pipeline development.
• Swedish Orphan Biovitrum (Sobi): Swedish rare-disease specialty pharma with a portfolio spanning genetic, metabolic, and pediatric rare diseases, leveraging centralized European regulatory approvals. Comparable to Gen.Orph in EMA-driven orphan-drug commercialization across Europe, the Middle East, and select emerging markets.
• Recordati Rare Diseases (Recordati Group): European specialty pharma division of Recordati that develops, registers, and commercializes orphan drugs across multiple rare disease areas. Directly comparable to Gen.Orph in business model (orphan drug focus), EMA-centric regulatory strategy, and multi-country distribution of rare-disease therapeutics for small patient populations.

Emerging players

• Inventiva: French clinical-stage biopharma developing therapies for rare metabolic diseases and fibrotic conditions. Comparable to Gen.Orph as a France-headquartered rare-disease therapeutics company targeting metabolic indications, although at earlier commercial stage with pipeline assets rather than Gen.Orph's commercial portfolio.

Regional players

• Stallergenes Greer: France-based specialty pharma with a focused product portfolio in allergy/rare immunology, owned by Nestlé Health Science. Comparable as a French-headquartered specialty pharma distributing niche products through established European and international channels, similar in operating geographies and regulatory registration approach to Gen.Orph.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/gen.orph

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds1 record

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Gen.Orph