Pharming Group

Name

Pharming Group

Legal name

Pharming Group N.V.

Website

https://www.pharming.com

Company type

Public

Founded year

1988

Operating status

Operating

Headcount range

251–500 employees

Short description

Pharming Group N.V. is a Dutch commercial-stage biopharmaceutical company that develops and commercializes protein replacement therapies and precision medicines for rare and ultra-rare immunological and genetic diseases, with two approved products: RUCONEST for hereditary angioedema and Joenja for APDS.

Ownership category

akta.pro rank

Product category

Rare disease biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Immunology, Autoimmune & Autoinflammatory Therapies (HLAIAIAE)

akta.pro secondary industries

Plasma-Derived Protease Inhibitors & Complement Modulators (e.g., C1 Esterase Inhibitor) (HLAIAHAF), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Rare disease therapeutics
• Protein replacement therapy
• Hereditary angioedema treatment
• Primary immunodeficiency medicines
• Biopharmaceutical commercialization

Headquarters

HQ city

Leiden

HQ country

Netherlands

HQ region

Europe

Offices4 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. RUCONEST Product Sales: RUCONEST is the company's legacy product for acute hereditary angioedema (HAE) attacks, generating record sales. The product is commercialized directly in the US, EU, and UK through Pharming's own sales organization and through distribution networks internationally. Revenue from RUCONEST reached US$317.9 million in FY2025.
2. Joenja Product Sales: Joenja (leniolisib) is the company's newer growth driver for APDS, approved in the US, UK, Japan, Australia, Israel, and now EU. Revenue from Joenja reached US$58.2 million in FY2025 and US$14.1 million in Q1 2026.
3. Licensing and Royalty Revenue: Pharming receives licensing fees and royalties from commercialization partners including OrphanPacific for Japan and NewBridge Pharmaceuticals for Middle East and North Africa.

Go-to-market motion3 records

Distribution channels4 records

Marketing channels5 records

Core offering

Pharming develops, manufactures, and commercializes protein replacement therapies and precision medicines for rare and ultra-rare immunological and genetic diseases. Its two commercial products are RUCONEST, a recombinant human C1 esterase inhibitor for acute hereditary angioedema attacks, and Joenja (leniolisib), an oral PI3K delta inhibitor for activated PI3K delta syndrome (APDS), with a pipeline extending into primary mitochondrial disease (napazimone/KL1333) and gene therapy for HAE (OTL-105).

Product overview

Pharming Group is an integrated biotechnology company with two commercial products: RUCONEST® (a recombinant human C1 esterase inhibitor for acute hereditary angioedema attacks) and Joenja® (leniolisib, a PI3Kδ inhibitor for APDS). The company has an advancing pipeline including napazimone (KL1333) for primary mitochondrial diseases and leniolisib expansion into CVID, acquired through the Abliva AB acquisition. The portfolio is focused on rare and ultra-rare immunological and genetic diseases.

Differentiator

Problem solved

Functional benefit

Brands

• RUCONEST: Recombinant human C1 esterase inhibitor (rhC1INH) protein replacement therapy for acute hereditary angioedema (HAE) attacks
• Joenja

Products and services

• RUCONEST Recombinant human C1 esterase inhibitor (rhC1INH) protein replacement therapy indicated for the treatment of acute hereditary angioedema (HAE) attacks in adults and adolescents. It is the first and only plasma-free rhC1INH protein replacement therapy, replacing deficient C1-INH to downregulate the complement and contact cascades that drive acute swelling episodes.
• Joenja (leniolisib) Oral phosphoinositide 3-kinase delta (PI3K delta) inhibitor indicated for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency caused by genetic mutations in the PI3K delta pathway. Joenja targets the underlying disease driver of APDS to reduce immune dysregulation and prevent complications such as recurrent infections and lymphoproliferation. Worldwide rights were licensed in from Novartis in 2019.
• Napazimone (KL1333) Investigational compound for the treatment of primary mitochondrial diseases (PMD), currently in the pivotal FALCON trial phase. Acquired through the February 2025 Abliva AB acquisition as a core mitochondrial medicine asset.
• Leniolisib (Phase II expansion) PI3K delta inhibitor in Phase II clinical trials as a pipeline expansion of leniolisib into Common Variable Immunodeficiency (CVID) and other primary immunodeficiencies beyond APDS.
• OTL-105 Investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of hereditary angioedema (HAE), developed in collaboration with Orchard Therapeutics under a strategic research, development, manufacturing, and commercialization agreement.

Quantifiable outcome

• Phase II/III trial met both primary endpoints with statistically significant improvements in immune markers in 31 APDS patients
• +2 more outcomes

Named customers4 records

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Five partnerships are on record, tiered core, regional, pipeline and market development.

• OrphanPacific, Inc.coreChannel Partner/ Reseller/ Distributor · 24 March 2026OrphanPacific commercializes Joenja (leniolisib) in Japan under a licensing agreement. Following Japan's approval of Joenja for APDS in patients aged 4 years and older, OrphanPacific handles commercialization including National Health Insurance drug pricing discussions. This is a key partnership for Pharming's expansion into the Japanese market.
• SanoficoreStrategic or Co-development Partner · 4 January 2022Sanofi and Pharming renewed their strategic manufacturing partnership agreement. Sanofi provides manufacturing capabilities for Pharming's products, including RUCONEST. This partnership ensures reliable supply chain and manufacturing capacity for the company's commercial products.
• NewBridge PharmaceuticalsregionalChannel Partner/ Reseller/ Distributor · 20 July 2021NewBridge Pharmaceuticals commercializes RUCONEST in the Middle East and North Africa region. This partnership extends Pharming's geographic reach into MEA markets where the company does not maintain direct commercial operations.
• Orchard TherapeuticspipelineStrategic or Co-development Partner · 1 July 2021Strategic collaboration to research, develop, manufacture, and commercialize OTL-105, an investigational ex vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema. The collaboration combines Orchard Therapeutics' gene therapy expertise with Pharming's rare disease commercial infrastructure.
• Invitae Corporationmarket developmentStrategic or Co-development Partner · 2 March 2021Pharming collaborated with Invitae to launch the navigateAPDS genetic testing program, helping patients get diagnosed with APDS. The program provides genetic testing to identify patients with the rare immunodeficiency, supporting disease awareness and patient identification for Joenja treatment.

Scale indicators11 records

Recent moves8 records

Expansion highlights6 records

Direct peers

• Swedish Orphan Biovitrum (Sobi): Sobi is a Sweden-based rare-disease biopharmaceutical company with overlapping HAE franchise (Kineret, other immunomodulatory therapies) and an established rare-disease commercial infrastructure across US and EU, making it directly comparable in target disease areas and business model.
• CSL Behring: CSL Behring markets Haegarda and Berinert, plasma-derived C1 esterase inhibitor products for HAE, directly competing with RUCONEST. As the leading plasma-protein therapeutics company, CSL Behring is a highly relevant direct competitor in Pharming's core market.
• BioCryst Pharmaceuticals: BioCryst markets Orladeyo, an oral prophylactic HAE therapy, directly competing with Pharming's HAE franchise. Its focus on rare-disease oral therapeutics and global commercialization make it a directly comparable competitor.

Broad incumbents

• Takeda Pharmaceutical: Takeda markets Takhzyro and Firazyr for HAE, directly competing with RUCONEST in the same therapeutic area, and operates a global rare-disease commercial organization including rare immunology and genetic disease portfolios.
• BioMarin Pharmaceutical: BioMarin is a leading rare-disease biopharma with a similar business model focused on genetic and ultra-rare conditions, comparable commercial-stage revenue scale, and a pipeline strategy emphasizing orphan indications with high pricing power.
• Recordati: Recordati Rare Diseases division markets treatments for rare conditions and serves as a relevant broad incumbent in the European rare-disease commercial space, with comparable focus on specialty/rare disease therapeutics.
• Alexion / AstraZeneca Rare Diseases: Alexion (now part of AstraZeneca) pioneered the rare-disease commercial model in complement-mediated diseases and serves as a broad incumbent benchmark for rare-disease commercial scale, infrastructure, and orphan-drug portfolio strategy.

Emerging players

• Alnylam Pharmaceuticals: Alnylam develops RNAi therapeutics for rare genetic diseases, including an HAE program, and operates in the same rare-disease commercial space with comparable specialty pharmaceutical infrastructure and orphan-drug pricing economics.
• argenx: argenx develops antibody therapeutics for rare autoimmune diseases, sharing Pharming's rare immunology focus and specialty commercialization model. The company represents a comparable emerging player in rare immune-mediated disease therapeutics.
• Ultragenyx Pharmaceutical: Ultragenyx develops treatments for rare and ultra-rare genetic diseases with a similar orphan-drug commercial model. Its focus on genetic and metabolic rare diseases makes it comparable in pipeline strategy and addressable patient population approach.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks7 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/pharming-healthcare-inc
• Xx.com/PharmingGroupNV

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Subsidiaries3 records

Funding overview

Funding rounds2 records

Investors3 records

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

M&A1 record

Investments

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

Frequently asked questions about Pharming Group