Grupo de Farming N.V. (PHAR): Modelo de Negócios Canvas [Jan-2025 Atualizado]

No mundo intrincado da biotecnologia, o grupo de farmões N.V. surge como uma força pioneira, transformando o tratamento de doenças raras por meio de terapias genéticas inovadoras. Sua tela de modelo de negócios meticulosamente criada revela uma abordagem sofisticada para enfrentar desafios médicos não atendidos, misturando pesquisas científicas de ponta com parcerias estratégicas e soluções personalizadas de saúde. Ao focar nas terapias de reposição enzimática e alavancar as tecnologias de engenharia genética proprietária, a farmagem não está apenas desenvolvendo medicamentos, mas criando esperança para pacientes com distúrbios genéticos complexos.

Pharming Group N.V. (PHAR) - Modelo de negócios: Parcerias -chave

Colaboração estratégica com centros médicos acadêmicos

O Grupo de Pharming N.V. mantém parcerias estratégicas com vários centros médicos acadêmicos de pesquisa e desenvolvimento clínico:

Instituição Acadêmica	Foco em parceria	Ano estabelecido
Centro Médico da Universidade de Leiden	Pesquisa de doenças raras	2018
Universidade de Amsterdã	Desenvolvimento da terapia genética	2020

Acordos de licenciamento com instituições de pesquisa farmacêutica

A Pharming estabeleceu acordos críticos de licenciamento com as seguintes instituições de pesquisa:

• Universidade de Michigan - Licença de pesquisa hereditária angioedema (HAE)
• Hospital Infantil da Filadélfia - Pesquisa de Transtorno Genético Raro
• Universidade de Stanford - colaboração de engenharia de proteínas

Parcerias de fabricação com empresas especializadas de biotecnologia

Empresa parceira	Capacidade de fabricação	Valor do contrato
Lonza Group AG	Produção de proteínas	€ 12,5 milhões de contrato anual
Wuxi Biologics	Fabricação de proteínas recombinantes	8,3 milhões de € Contrato anual

Redes de pesquisa colaborativa em desenvolvimento de tratamento de doenças raras

Principais colaborações da rede de pesquisa:

• Consórcio europeu de pesquisa de doenças raras
• Rede Internacional de Angioedema
• Aliança de Pesquisa de Distúrbios Genéticos Globais

Investimento total de parceria em 2023: € 37,6 milhões

Pharming Group N.V. (PHAR) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de drogas de doenças raras

O Grupo de Farming N.V. concentra -se em pesquisas terapêuticas de doenças raras, com ênfase específica no angioedema hereditário (HAE) e em outros distúrbios genéticos raros. A empresa investiu € 25,8 milhões em despesas de pesquisa e desenvolvimento em 2022.

Área de foco de pesquisa	Investimento (2022)	Programas de pesquisa ativa
Distúrbios genéticos raros	€ 25,8 milhões	3 programas primários

Inovação da terapia de reposição enzimática

Especializado no desenvolvimento da terapêutica proteica recombinante, com foco primário nas estratégias de substituição de enzimas.

• RUCONEST® (inibidor da esterase C1) como produto de substituição da enzima primária
• Desenvolvimento contínuo de novas terapias enzimáticas
• Plataformas proprietárias de tecnologia de produção de proteínas

Gerenciamento e execução de ensaios clínicos

Fase de ensaios clínicos	Número de ensaios ativos (2023)	Investimento total
Fase I/II	2 ensaios	€ 8,5 milhões
Fase III	1 Trial	€ 12,3 milhões

Processos de conformidade regulatória e registro de drogas

Estratégia regulatória abrangente em várias jurisdições, incluindo mercados da UE, EUA e Global.

• Instalações de fabricação registradas pela FDA
• Protocolos de conformidade EMA
• Submissões regulatórias ativas em 3 principais mercados

Fabricação especializada de produtos farmacêuticos

Capacidades avançadas de fabricação biofarmacêutica com tecnologias especializadas de produção de proteínas.

Capacidade de fabricação	Volume anual de produção	Padrão de controle de qualidade
Produção de proteínas transgênicas	Até 500 kg/ano	Certificado GMP

Pharming Group N.V. (PHAR) - Modelo de negócios: Recursos -chave

Tecnologias de engenharia genética proprietária

O Grupo de Farming N.V. utiliza plataformas avançadas de engenharia genética projetadas especificamente para a produção de proteínas em animais transgênicos. A principal plataforma tecnológica da empresa envolve:

• Tecnologia transgênica de coelho para produção de proteínas recombinantes
• Técnicas de modificação genética de precisão
• Processos de purificação de proteínas proprietários

Categoria de tecnologia	Plataforma específica	Status de desenvolvimento
Engenharia genética	Plataforma transgênica de coelho	Totalmente operacional
Produção de proteínas	Extração de proteínas biofarmacêuticas	Tecnologia validada

Equipes de pesquisa científica especializadas

A Pharming mantém equipes de pesquisa altamente especializadas com experiência em doenças raras e biotecnologia.

• Pessoal Total de Pesquisa: 132 funcionários (a partir de 2023)
• Pesquisadores no nível de doutorado: 47
• Locais de pesquisa: Holanda, Estados Unidos

Portfólio de propriedade intelectual

A propriedade intelectual da Pharming representa um recurso -chave crítico para a empresa.

Categoria IP	Número de patentes	Cobertura geográfica
Patentes concedidas	38	Europa, Estados Unidos, Internacional
Aplicações de patentes pendentes	12	Múltiplas jurisdições

Instalações avançadas de pesquisa de biotecnologia

A Pharming opera instalações de pesquisa e produção de ponta.

• Espaço total da instalação de pesquisa: 4.500 metros quadrados
• Biossegurança Nível 2 e 3 Laboratórios
• Complexo de pesquisa e fabricação integrada em Leiden, Holanda

Extensa experiência em tratamento de doenças raras

A empresa se concentra no desenvolvimento de tratamentos para distúrbios genéticos raros.

Área terapêutica	Programas de tratamento atuais	Estágio clínico
Angioedema hereditário	Ruconest (inibidor recombinante da esterase C1)	Aprovado e comercializado
Outras doenças raras	Vários programas de investigação	Vários estágios de desenvolvimento

Grupo de Farming N.V. (PHAR) - Modelo de negócios: proposições de valor

Tratamentos inovadores para distúrbios genéticos raros

O Grupo de Farming N.V. se concentra no desenvolvimento de terapias para distúrbios genéticos raros, direcionando -se especificamente:

Transtorno	Produto	Estágio de desenvolvimento	Potencial de mercado
Angioedema hereditário (hae)	RUCONEST	Aprovado	Mercado global de US $ 500 milhões
Doença de Fabry	PRN1006	Fase de ensaios clínicos	Mercado potencial de US $ 1,2 bilhão

Terapias de reposição enzimática direcionadas

As terapias de reposição enzimática desenvolvidas por farmings incluem:

• Substituição do inibidor da esterase C1 para HAE
• Terapias de proteínas recombinantes
• Tratamentos enzimáticos de engenharia de precisão

Tipo de terapia	Receita anual (2023)	Taxa de crescimento
RUCONEST	€ 111,4 milhões	18.4%
Substituição enzimática	€ 132,6 milhões	22.7%

Soluções médicas personalizadas para necessidades não atendidas do paciente

Abordagem da Pharming para soluções médicas personalizadas:

• Tratamentos específicos do distúrbio genético
• Desenvolvimento de medicamentos centrado no paciente
• Estratégias de medicina de precisão

Intervenções biotecnológicas avançadas

As capacidades biotecnológicas incluem:

• Plataforma de produção de proteínas proprietária
• Tecnologia de proteínas recombinantes
• Pesquisa de terapia genética

Investimento em P&D	2023 quantidade	Porcentagem de receita
Despesas de pesquisa	€ 37,2 milhões	28.1%

Produtos terapêuticos de alta qualidade com eficácia clínica comprovada

Métricas de desempenho clínico:

Produto	Taxa de sucesso clínico	Satisfação do paciente
RUCONEST	92.3%	87%
Terapias enzimáticas	89.7%	85%

Grupo de Farming N.V. (PHAR) - Modelo de Negócios: Relacionamentos do Cliente

Médico direto e engajamento do profissional de saúde

O Pharming Group N.V. mantém o envolvimento direto com 782 prestadores de serviços de saúde especializados em 14 países a partir do quarto trimestre de 2023. A equipe de assuntos médicos da empresa conduz 213 eventos de educação médica direcionados anualmente.

Métrica de engajamento	2023 dados
Total de prestadores de serviços de saúde envolvidos	782
Eventos de educação médica	213
Países cobertos	14

Programas de apoio ao paciente e educação

A farmagem opera programas abrangentes de apoio ao paciente com 1.647 matrículas ativas de pacientes em 2023.

• Helpline de apoio ao paciente 24 horas por dia, 7 dias por semana
• Portal de Recursos de Paciente Digital
• Programas de assistência financeira

Métrica do Programa de Pacientes	2023 Estatísticas
Matrículas ativas em pacientes	1,647
Apoie o tempo de resposta da linha de apoio	<15 minutos

Serviços personalizados de consulta médica

Farming fornece Serviços especializados de aconselhamento genético com 437 consultas individuais realizadas em 2023.

Plataformas de informações de saúde digital

A empresa mantém uma plataforma digital com 92.413 usuários registrados em dezembro de 2023.

Métrica da plataforma digital	2023 dados
Usuários registrados	92,413
Usuários ativos mensais	41,276

Acompanhamento clínico e monitoramento em andamento

A farmings rastreia 2.316 pacientes por meio de programas de monitoramento clínico de longo prazo em 2023.

• Check-ins trimestrais dos pacientes
• Integração eletrônica de registro de saúde
• Rastreamento de tratamento personalizado

Métrica de monitoramento clínico	2023 Estatísticas
Pacientes totais monitorados	2,316
Frequência de monitoramento	Trimestral

Pharming Group N.V. (PHAR) - Modelo de Negócios: Canais

Vendas diretas para centros médicos especializados

O Pharming Group N.V. vende diretamente seus produtos farmacêuticos a centros médicos especializados por meio de estratégias de vendas direcionadas.

Tipo de canal	Número de centros médicos	Cobertura geográfica
Centros de tratamento de doenças raras	127	América do Norte, Europa, Austrália
Hemofilia Clínicas especializadas	84	Estados Unidos, União Europeia

Redes de distribuição farmacêutica

A farmagem utiliza extensas redes de distribuição farmacêutica para atingir os profissionais de saúde e pacientes.

• Distribuidores farmacêuticos primários: Amerisourcebergen, Cardinal Health
• Parcerias de distribuição internacional: 6 principais atacadistas farmacêuticos
• Alcance de distribuição global: 18 países

Plataformas de informações médicas online

Canais digitais para disseminação de informações médicas e comunicação do produto.

Tipo de plataforma	Visitantes únicos mensais	Foco de conteúdo
Pharming Site Corporate	42,500	Informações do produto, pesquisa clínica
Portal profissional médico	23,750	Dados científicos, protocolos de tratamento

Apresentações da conferência médica

As conferências científicas servem como canais críticos para visibilidade do produto e compartilhamento de pesquisas.

• Conferências anuais participaram: 12
• Tipos de conferência: doenças raras, hematologia, distúrbios genéticos
• Frequência de apresentação: 8-10 apresentações científicas por ano

Publicação científica e disseminação de pesquisa

As farmings aproveitam as publicações acadêmicas e médicas para comunicar os avanços científicos.

Categoria de publicação	Número de publicações (2023)	Faixa de fatores de impacto
Revistas revisadas por pares	14	2.5 - 8.7
Resumos de pesquisa	22	N / D

Pharming Group N.V. (PHAR) - Modelo de negócios: segmentos de clientes

Pacientes com doenças raras

O Grupo de Farming N.V. tem como alvo principalmente pacientes com distúrbios genéticos raros específicos, especificamente:

• Pacientes com angioedema hereditário (HAE): aproximadamente 1 em 50.000 indivíduos em todo o mundo
• Pacientes com deficiência de antitripsina alfa-1: prevalência global estimada de 1 em 2.500 indivíduos

Doença rara	População global de pacientes	Valor de mercado de tratamento
Angioedema hereditário	10.000-15.000 pacientes diagnosticados nos Estados Unidos	US $ 2,1 bilhões até 2026
Deficiência de antitripsina alfa-1	100.000 pacientes diagnosticados nos Estados Unidos	US $ 1,5 bilhão até 2025

Médicos especializados

Especialistas médicos -alvo incluem:

• Imunologistas
• Geneticistas
• Hematologistas
• Pulmonologistas

Centros de Tratamento de Transtornos Genéticos

Pharming metas de alvos de centros de tratamento especializados em:

• Estados Unidos: 250 Centros de Tratamento de Transtornos Genéticos Especializados
• União Europeia: 180 Centros de Tratamento de Transtornos Genéticos Especializados
• Região da Ásia-Pacífico: 120 Centros de Tratamento de Transtornos Genéticos Especializados

Provedores de seguros de saúde

Região	Número de provedores de seguros	Taxa de cobertura de doenças raras
Estados Unidos	150 principais provedores de seguros	72% de cobertura para tratamentos de doenças raras
União Europeia	95 principais provedores de seguros	68% de cobertura para tratamentos de doenças raras

Instituições de pesquisa

As farmings colaboram com instituições de pesquisa com foco em terapias genéticas:

• Estados Unidos: 85 instituições de pesquisa
• União Europeia: 62 instituições de pesquisa
• Parcerias de Pesquisa Global: 15 colaborações ativas

Foco na pesquisa	Número de instituições	Financiamento anual de pesquisa
Doenças raras genéticas	47 instituições	Financiamento anual total de US $ 350 milhões
Terapias de reposição de proteínas	38 instituições	US $ 275 milhões totais de financiamento anual

Grupo de Farming N.V. (PHAR) - Modelo de negócios: Estrutura de custos

Extensos investimentos em pesquisa e desenvolvimento

Em 2022, o Grupo de Pharming N.V. relatou despesas de P&D de € 57,2 milhões, representando 35,4% do total de despesas operacionais.

Ano	Despesas de P&D (milhões de euros)	Porcentagem de despesas operacionais
2022	57.2	35.4%
2021	46.8	33.2%

Despesas de ensaios clínicos

Os custos de ensaios clínicos para os principais desenvolvimentos do produto RUCONEst® e do oleoduto da Pharming foram estimados em 22,5 milhões de euros em 2022.

• Ensaios clínicos de fase III para tratamentos de doenças raras
• Programas de pesquisa em doenças genéticas em andamento
• Despesas contínuas de recrutamento e monitoramento de pacientes

Custos de fabricação e produção

As despesas de fabricação de 2022 totalizaram aproximadamente € 38,6 milhões.

Categoria de custo	Valor (milhões de euros)
Custos de matéria -prima	15.3
Manutenção da instalação de produção	8.7
Controle de qualidade	6.2
Depreciação do equipamento	8.4

Despesas de conformidade regulatória

Os custos de conformidade regulatória para 2022 foram de aproximadamente 12,4 milhões de euros.

• Taxas de envio da FDA e EMA
• Documentação regulatória em andamento
• Sistemas de monitoramento de conformidade

Manutenção da propriedade intelectual

As despesas de propriedade intelectual em 2022 atingiram € 5,6 milhões.

Categoria de despesa IP	Valor (milhões de euros)
Registro de patentes	2.3
Manutenção de patentes	1.8
Proteção legal	1.5

Pharming Group N.V. (PHAR) - Modelo de negócios: fluxos de receita

Vendas farmacêuticas de produtos

Em 2022, o Grupo de Farming N.V. relatou receita total de 224,7 milhões de euros, com receita primária gerada a partir de vendas da Ruconest® (inibidor da esterase C1).

Produto	Receita (2022)	Segmento de mercado
RUCONEST®	224,7 milhões de euros	Tratamento hereditário de angioedema

Acordos de licenciamento

A Pharming possui parcerias estratégicas de licenciamento gerando fluxos de receita adicionais.

• Contrato de licenciamento com a Horizon Therapeutics for Ruconest®
• Licenciamento de licenciamento com a CSL Behring for Global Rights

Financiamento de colaboração de pesquisa

O financiamento de colaboração de parcerias de pesquisa contribuiu com 12,5 milhões de euros em 2022.

Subsídios governamentais e institucionais

Recebeu subsídios de pesquisa totalizando aproximadamente € 3,2 milhões em 2022 para pesquisa de doenças raras.

Renda de royalties de tecnologias patenteadas

Receita de royalties de tecnologias farmacêuticas proprietárias estimadas em 5,6 milhões de euros em 2022.

Fluxo de receita	Valor (2022)
Vendas de produtos	224,7 milhões de euros
Acordos de licenciamento	Não revelado
Colaboração de pesquisa	€ 12,5 milhões
Subsídios do governo	€ 3,2 milhões
Renda de royalties	€ 5,6 milhões

Pharming Group N.V. (PHAR) - Canvas Business Model: Value Propositions

You're looking at the core value Pharming Group N.V. delivers to its customers-the patients and prescribers in the ultra-rare disease space. It's all about providing specialized, life-altering treatments where few other options exist. This is where the company truly earns its keep.

On-Demand HAE Treatment: RUCONEST®

For Hereditary Angioedema (HAE) patients needing acute attack treatment, Pharming Group N.V. offers RUCONEST®, a recombinant C1-INH protein replacement therapy. This product is positioned as the on-demand treatment that targets the root cause via IV administration, which means rapid onset. Honestly, the numbers show its continued strength in the U.S. market, even with new oral competition.

Here's a quick look at how RUCONEST® performed in the third quarter of 2025:

Metric	Value (Q3 2025)	Comparison
Revenue	US$82.2 million	+29% versus Q3 2024
U.S. Volume Growth	+24%	In the third quarter of 2025
U.S. Volume Growth (YTD)	+28%	For the first nine months of 2025
Acute Attack Efficacy	97%	Attacks treated with just 1 dose
Sustained Efficacy	93%	Acute attacks stopped for at least 3 days

The strategic move to withdraw RUCONEST® from non-US markets underscores the focus on maximizing value where the demand and infrastructure are most robust, which is definitely the U.S. base.

Oral APDS Treatment: Joenja® (Leniolisib)

Joenja® is a major value driver, being the first and only approved oral treatment for Activated PI3K-delta Syndrome (APDS) in the 12 years and older segment. You see the acceleration in uptake reflected clearly in the financials as more patients get diagnosed and transitioned to paid therapy.

The growth story for Joenja® in the third quarter of 2025 is quite compelling:

• Revenue reached US$15.1 million, a 35% year-over-year increase.
• Unit sales volume grew by 34% in the third quarter of 2025.
• The U.S. patient base on paid therapy reached 116 patients, up 25% year-over-year.
• The company added 13 new APDS patients on therapy in the U.S. during Q3 2025 alone.
• Joenja® revenue for the first nine months of 2025 was US$38.4 million.

Also, the potential market is expanding; the FDA granted priority review for the pediatric indication (ages 4 to 11), with a decision expected by January 2026.

Pipeline Targeting High Unmet Need

Pharming Group N.V.'s pipeline extends its value proposition into other ultra-rare diseases, leveraging the expertise gained from APDS. This is about future-proofing the revenue base with high-impact science.

For APDS, new research published in Cell suggests that reclassifying patients with a variant of uncertain significance (VUS) could imply up to a 100-fold increase in APDS prevalence, opening up a much larger addressable patient pool. Beyond that, the company has an additional 180 APDS patients in access programs and clinical studies globally.

For mitochondrial disease, the company is actively recruiting for the pivotal FALCON clinical trial studying leniolisib in mitochondrial DNA-driven primary mitochondrial diseases. The current cash position, which stood at US$168.9 million at the end of Q3 2025, is expected to cover these pipeline and pre-launch costs.

Patient-Centric Support

The value proposition is cemented by support programs that ensure continuity of care, which is critical in rare disease management. This is evidenced by the strong adherence rates for Joenja® and the continued growth in U.S. prescribers for RUCONEST®.

The company's overall 2025 total revenue guidance was raised to a range of US$365 million to US$375 million, reinforcing the market's acceptance of the value delivered by these specialized therapies.

Pharming Group N.V. (PHAR) - Canvas Business Model: Customer Relationships

You're looking at how Pharming Group N.V. maintains its connection with the specialized prescribers and the patients relying on its niche, often lifelong, therapies. This isn't about mass-market advertising; it's about deep, focused engagement in the rare disease space.

Dedicated Rare Disease Sales Force: Direct engagement with specialized prescribers.

• The commercial strategy relies heavily on direct engagement with specialists.
• The U.S. market is the core focus, contributing 97% of first quarter 2025 revenues.
• Sustained growth in RUCONEST® reflects a consistent addition of prescribers.
• Pharming Group N.V. added an average of 22 new prescribers over the past six quarters leading up to the third quarter of 2025.
• For Joenja®, there was a 25% year-over-year increase in patients on paid therapy in the third quarter of 2025.

The relationship with the prescribing community is clearly driving volume, as seen in the revenue performance for their two key assets:

Metric	Time Period	Value/Amount
RUCONEST® Revenue Growth (YoY)	Q3 2025	29%
Joenja® Revenue Growth (YoY)	Q3 2025	35%
RUCONEST® Revenue Growth (YoY)	First Nine Months 2025	34%
Joenja® Patients on Paid Therapy Growth (YoY)	Q3 2025	25%

High-touch patient support programs (e.g., Ruconest support line).

For on-demand treatments like RUCONEST®, efficacy in the moment is paramount, which necessitates strong post-sale support and clear clinical data to reinforce trust.

• RUCONEST® is mostly used by patients experiencing more severe/frequent attacks.
• In a comparative real-world study, 97% of HAE attacks treated with RUCONEST® were resolved after the first dose.
• Another study indicated 93% of acute attacks were stopped for at least 3 days following treatment.

Educational campaigns for immunologists and healthcare providers (HCPs).

The focus here is on establishing the product's unique value proposition, especially against newer competition, which requires ongoing education.

• The sustained success of RUCONEST® is attributed to its unique profile and strong differentiation in the on-demand HAE market, even after a new oral acute product launched in July 2025.
• For Joenja®, the company is preparing to file for U.S. FDA approval for pediatrics in the third quarter of 2025.
• Pharming Group N.V. continues to work closely with global patient organizations.

Long-term relationships with patients due to chronic, lifelong treatments.

The nature of treating conditions like Hereditary Angioedema (HAE) and APDS (Activated PI3K-delta Syndrome) means the relationship is inherently long-term, often spanning the patient's life.

• As of June 30, 2025, Pharming Group N.V. had identified 971 diagnosed APDS patients globally.
• Of those, 257 patients were identified in the U.S..
• For Joenja® in the U.S., patient numbers on paid therapy grew from 91 at the end of Q2 2024 to 114 at the end of Q2 2025.
• The company expects to continue to identify and enroll new APDS patients, supported by VUS (Variant of Uncertain Significance) resolution efforts.

Finance: review Q4 2025 patient enrollment projections against the current sales force deployment by next Tuesday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Channels

You're looking at how Pharming Group N.V. gets its therapies, like RUCONEST® and Joenja®, from the lab to the patient, which is a critical part of their rare disease strategy. For orphan drugs, the channel is never just a simple drop-ship; it's highly specialized and relationship-driven.

Specialty Pharmacies and Distributors: Direct distribution of orphan drugs.

Pharming Group N.V. relies on a specialized distribution network, which is typical for high-cost, low-volume orphan drugs. The global specialty drug distribution market was valued at approximately USD 298.48 billion in 2024, indicating the complex environment they operate within. For Pharming Group N.V., the channel focus is heavily skewed geographically, with the U.S. market driving the vast majority of sales. In the second quarter of 2025, the U.S. market accounted for 92% of total revenues, leaving only 8% from the EU and the Rest of World. This concentration means channel management is intensely focused on securing access through key specialty pharmacies and distributors capable of handling these specific therapies, often within limited or exclusive networks.

Direct Sales Teams: Targeting US and European HAE/Immunology specialists.

The sales channel is built around specialists who educate prescribers on Hereditary Angioedema (HAE) and Activated PI3K Delta Syndrome (APDS). The growth in revenue reflects the success of these teams in increasing prescribers. For instance, RUCONEST® second quarter 2025 revenue grew by 28% year-over-year, reflecting strong growth in patients and prescribers. The sales effort is clearly weighted toward the U.S., which is the primary revenue engine. The company is actively managing its cost structure, announcing a plan to reduce general and administrative (G&A) expenses by 15% or US$10 million in 2025 to optimize capital allocation, which would impact the size and efficiency of these teams.

Digital Patient Tools: Joenja patient app for adherence and support.

For Joenja, patient support is integrated directly into the channel to drive adherence and manage the complex journey from diagnosis to paid therapy. While specific patient app usage numbers aren't public, the acceleration in patient uptake is clear. As of June 30, 2025, Pharming Group N.V. had 114 patients on paid Joenja therapy in the U.S., a 25% increase from the prior year's second quarter. The company supports this with the APDS Assist program, which helps enroll patients and navigate insurance requirements. The first half of 2025 saw Joenja patient growth surpass the total increase for all of 2024.

Regulatory Filings: Securing market access via FDA, EMA, NICE, and TGA approvals.

Regulatory success is a prerequisite for channel activation in new markets. Pharming Group N.V. has made significant progress in securing market access throughout 2025:

• Joenja launched in England and Wales in April 2025 after NICE positive final guidance.
• TGA approval was secured in Australia in the first quarter of 2025.
• A supplemental New Drug Application (sNDA) for pediatric Joenja use (ages 4 to 11) was submitted to the U.S. FDA in the third quarter of 2025.
• The EMA review for APDS patients 12 years and older is on track, with a response due by the January 2026 deadline.
• A New Drug Application for leniolisib was submitted in Japan.

Here's a quick look at the channel performance metrics as of mid-2025:

Metric	Value/Period	Source Context
Q3 2025 Total Revenue	US$97.3 million	Third Quarter 2025 Results
Q3 2025 Joenja Revenue	US$15.1 million	Third Quarter 2025 Results
US Patients on Paid Joenja Therapy (as of Q2 2025)	114 patients	Second Quarter 2025 Results
US Market Revenue Contribution (Q2 2025)	92%	Second Quarter 2025 Results
Total Diagnosed APDS Patients Globally (as of Q2 2025)	971 patients	Second Quarter 2025 Results
2025 Total Revenue Guidance (Raised July 2025)	US$335 million - US$350 million	Second Quarter 2025 Results

If onboarding takes 14+ days, churn risk rises, which is why the APDS Assist program is defintely a key part of the channel support structure. Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Customer Segments

You're looking at the core patient populations Pharming Group N.V. serves as of late 2025, which directly drives the revenue from their two key commercial assets, RUCONEST® and Joenja®. The focus is intensely specialized, targeting rare diseases where they hold or seek leading positions.

Hereditary Angioedema (HAE) patients requiring on-demand treatment

This segment is served by RUCONEST® (C1 esterase inhibitor, lyophilized for intravenous use), which remains the financial bedrock for Pharming Group N.V. The growth in this segment is evident in the top-line numbers. For the third quarter of 2025, RUCONEST® revenue hit US$82.2 million, marking a 29% increase compared to the third quarter of 2024. Looking at the longer trend, revenue for the first nine months of 2025 reached US$231.2 million, a 34% jump year-over-year. This sustained performance is fueled by growth in both the number of patients and the prescribers in the competitive U.S. HAE market. To be fair, this segment is facing new competition, as the company noted the launch of a new oral on-demand therapy in July 2025, but RUCONEST® still showed strong volume growth in the U.S. during that quarter.

Activated PI3K Delta Syndrome (APDS) patients in the US and EU

The customer base for Joenja® (leniolisib) is the population diagnosed with Activated PI3K Delta Syndrome (APDS). As of September 30, 2025, Pharming Group N.V. had identified 990 diagnosed APDS patients globally. The U.S. market is the primary focus for Joenja® uptake, with 270 patients identified there as of the end of Q3 2025. Of those U.S. patients, 175 were 12 years of age or older and eligible for Joenja® treatment. This segment is rapidly expanding its revenue contribution; Q3 2025 Joenja® revenue was US$15.1 million, a 35% increase year-over-year. For the first nine months of 2025, Joenja® generated US$38.4 million in revenue. The company is also actively working to reclassify patients with a Variant of Uncertain Significance (VUS) in the implicated genes, with over 1,400 such U.S. patients identified as of mid-2025.

Here's a quick look at the revenue contribution from these two rare disease segments for the third quarter of 2025:

Indication/Product	Q3 2025 Revenue (US$)	Year-over-Year Growth
HAE (RUCONEST®)	82.2 million	29%
APDS (Joenja®)	15.1 million	35%
Total Revenue	97.3 million	30%

Immunologists and Hematologists who treat rare immune deficiencies

These specialists are the key channel partners and prescribers. For the HAE segment, Pharming Group N.V. increased its RUCONEST® physician prescriber base by 11% during the full year 2024, adding many previously unknown HAE prescribers. This indicates a continuous effort to map and engage the treating specialists for HAE. For APDS, the growth in Joenja® revenue is driven by a 25% year-over-year increase in patients on paid therapy in the U.S. as of Q3 2025, which requires close collaboration with the specialized Immunologists and Hematologists who diagnose and manage APDS.

Pediatric patients (future segment) following planned Q3 2025 FDA filing for Joenja

This represents a significant near-term expansion opportunity for Joenja®. Pharming Group N.V. planned an FDA filing for children aged 1-11 years in the third quarter of 2025. The U.S. Food and Drug Administration (FDA) granted Priority Review for the supplemental New Drug Application (sNDA) for children aged 4 to 11 years, with a decision date set for January 31, 2026. As of September 30, 2025, there were 54 identified U.S. patients aged 4 to 11 who would become eligible upon regulatory approval. This group is about one-quarter of all APDS patients currently identified in the U.S.. Successful approval could unlock an additional €10-15 million in annual peak sales.

The overall confidence in the commercial trajectory, driven by these segments, led Pharming Group N.V. to raise its total 2025 revenue guidance to between US$365 million and US$375 million as of November 2025.

Pharming Group N.V. (PHAR) - Canvas Business Model: Cost Structure

You're looking at the core expenditures that fuel Pharming Group N.V.'s operations as of late 2025. This structure is heavily weighted toward supporting the commercialization of existing assets and advancing the late-stage pipeline, especially following the Abliva acquisition.

The overall spending envelope for the year is clearly defined. Pharming Group N.V. guided Total Operating Expenses for Fiscal Year 2025 between US$304 million and US$308 million. This guidance already factors in the costs associated with the Abliva acquisition, specifically noting an expected US$10.2 million in non-recurring Abliva-related transaction and integration expenses within that total operating expense figure.

The company is also planning for a one-time hit in the final quarter. Expect non-recurring restructuring costs of approximately $7 million to be recorded in Q4 2025. This is tied to an organizational restructuring that includes a 20% net reduction in non-commercial and non-medical headcount, primarily at the Netherlands headquarters.

Here's a look at the costs associated with producing the company's products, which are complex biologics and small molecules.

Cost Component	Q2 2025 Amount (US$m)	1H 2025 Amount (US$m)
Cost of Sales (COGS)	9.0	17.3

The Cost of Goods Sold, or Cost of Sales, for the second quarter of 2025 was US$9.0 million, bringing the total for the first half of 2025 to US$17.3 million. This reflects the costs inherent in manufacturing their specialized products like RUCONEST®, a complex biologic.

Research and Development (R&D) expenses are significant, driven by late-stage clinical work. The pivotal FALCON clinical trial, evaluating KL1333 for primary mitochondrial disease, is a major cost center. The total incremental cost related to KL1333, covering the purchase price, transaction/integration, and development costs, is estimated to be approximately US$133 million ahead of the expected FDA approval in 2028. For the first nine months of 2025, non-recurring Abliva acquisition-related expenses included US$2.1 million in R&D.

Sales, General, and Administrative (SG&A) costs support the global commercial teams for products like RUCONEST® and Joenja®. The company is actively managing these overheads. They remain on track to reduce total G&A expenses by 15% or US$10 million annually as part of the restructuring plan. Non-recurring Abliva acquisition-related expenses impacted SG&A as well, with US$8.0 million recorded in G&A for the first nine months of 2025.

You can see how the Abliva-related costs are distributed across the operating expense categories:

• Non-recurring Abliva acquisition-related expenses totaled US$10.2 million within the FY 2025 Operating Expenses guidance.
• For the first nine months of 2025, these non-recurring costs included US$8.0 million in G&A and US$2.1 million in R&D.
• In Q1 2025 alone, non-recurring Abliva costs included US$5.7 million in G&A and US$2.1 million in R&D.

The company is definitely making structural changes to manage its cost base going forward. Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Revenue Streams

You're looking at the core income sources for Pharming Group N.V. as of late 2025. The revenue picture is clearly defined by two primary pharmaceutical products, with the full-year outlook recently updated based on strong mid-year performance.

The revenue streams are built around the commercial success of its two main assets. Ruconest, for acute Hereditary Angioedema (HAE) attacks, continues to be a major cash generator, especially in the U.S. market, even with new competition entering the space. Joenja (leniolisib), for the treatment of APDS (Activated PI3K-delta Syndrome), is showing significant growth as patient uptake accelerates.

Here's a breakdown of the key financial figures driving the revenue streams as reported through the third quarter of 2025:

• - Ruconest product sales for acute HAE attacks.
• - Joenja (leniolisib) product sales for APDS.
• - Total 2025 revenue guidance is US$365 million - US$375 million.
• - Q3 2025 Ruconest revenue was US$82.2 million.
• - Q3 2025 Joenja revenue was US$15.1 million.

The third quarter of 2025 showed robust performance, leading Pharming Group N.V. to raise its full-year revenue expectations. The total revenue for the third quarter of 2025 hit US$97.3 million, a 30% increase compared to the third quarter of 2024. This momentum is what underpins the revised full-year guidance.

You can see the specific contributions from the two key products in the table below, which also includes the year-to-date performance for the first nine months of 2025.

Revenue Component	Q3 2025 Revenue (US$ million)	9M 2025 Revenue (US$ million)
RUCONEST	82.2	231.2
Joenja (leniolisib)	15.1	38.4

The growth in Joenja revenue, which increased by 35% in the third quarter compared to the prior year's third quarter, reflects strong growth in patients on paid therapy. Meanwhile, RUCONEST revenue grew by 29% year-over-year for the quarter, driven by sustained growth in both patients and prescribers in the HAE market. The company noted that RUCONEST is well positioned to provide continued strong cash flows. The revenue from other markets outside of the main focus areas contributed only US$1.1 million, or 1.3% of total RUCONEST revenue in the current quarter, and these markets have not shown financial sustainability.

The overall financial expectation for the year is clear:

• The 2025 total revenue guidance was raised to US$365 million - US$375 million, up from the prior guidance of US$335 million - US$350 million.
• The revenue for the first nine months of 2025 reached US$231.2 million from RUCONEST alone.

This revenue base is what management is using to fund pipeline opportunities and cover current operating expenses, which are projected between US$304 million and US$308 million for the full year 2025, before a one-time restructuring cost of approximately $7 million expected in the fourth quarter.