Pharming Group N.V. (PHAR): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el intrincado mundo de la biotecnología, el Grupo Farming N.V. emerge como una fuerza pionera, transformando el tratamiento de enfermedades raras a través de terapias genéticas innovadoras. Su lienzo de modelo de negocio meticulosamente elaborado revela un enfoque sofisticado para abordar los desafíos médicos no satisfechos, combinando la investigación científica de vanguardia con asociaciones estratégicas y soluciones de atención médica personalizadas. Al centrarse en las terapias de reemplazo de enzimas y aprovechar las tecnologías patentadas de ingeniería genética, la farmacia no solo está desarrollando medicamentos, sino que crea esperanza para pacientes con trastornos genéticos complejos.

Pharming Group N.V. (PHAR) - Modelo de negocio: asociaciones clave

Colaboración estratégica con centros médicos académicos

Pharming Group N.V. mantiene asociaciones estratégicas con varios centros médicos académicos para la investigación y el desarrollo clínico:

Institución académica	Enfoque de asociación	Año establecido
Centro Médico de la Universidad de Leiden	Investigación de enfermedades raras	2018
Universidad de Amsterdam	Desarrollo de terapia genética	2020

Acuerdos de licencia con instituciones de investigación farmacéutica

Pharming ha establecido acuerdos críticos de licencia con las siguientes instituciones de investigación:

• Licencia de investigación de la Universidad de Michigan - Angioedema hereditario (HAE)
• Hospital de niños de Filadelfia - Investigación de trastorno genético raro
• Universidad de Stanford - Colaboración de ingeniería de proteínas

Asociaciones de fabricación con compañías de biotecnología especializadas

Empresa asociada	Capacidad de fabricación	Valor de contrato
Grupo Lonza AG	Producción de proteínas	Contrato anual de € 12.5 millones
Wuxi Biologics	Fabricación de proteínas recombinantes	Acuerdo anual de 8,3 millones de euros

Redes de investigación colaborativa en desarrollo de tratamiento de enfermedades raras

Colaboraciones de redes de investigación clave:

• Consorcio europeo de investigación de enfermedades raras
• Red Internacional de Angioedema
• Alianza de investigación de trastornos genéticos globales

Inversión total de asociación en 2023: 37,6 millones de euros

Pharming Group N.V. (PHAR) - Modelo de negocio: actividades clave

Investigación y desarrollo de drogas de enfermedades raras

Pharming Group N.V. se centra en la investigación terapéutica de la enfermedad rara con énfasis específico en el angioedema hereditario (HAE) y otros trastornos genéticos raros. La compañía invirtió 25,8 millones de euros en gastos de investigación y desarrollo en 2022.

Área de enfoque de investigación	Inversión (2022)	Programas de investigación activos
Trastornos genéticos raros	25.8 millones de euros	3 programas primarios

Innovación de terapia de reemplazo enzimático

Especializado en el desarrollo de la terapéutica de proteínas recombinantes, con un enfoque primario en las estrategias de reemplazo de enzimas.

• Ruconest® (inhibidor de la esterasa C1) como producto de reemplazo de enzimas primario
• Desarrollo continuo de nuevas terapias enzimáticas
• Plataformas de tecnología de producción de proteínas patentadas

Gestión y ejecución del ensayo clínico

Fase de ensayo clínico	Número de ensayos activos (2023)	Inversión total
Fase I/II	2 pruebas	8,5 millones de euros
Fase III	1 juicio	12.3 millones de euros

Procesos de cumplimiento regulatorio y registro de medicamentos

Estrategia regulatoria integral en múltiples jurisdicciones, incluidos los mercados de la UE, EE. UU. Y Global.

• Instalaciones de fabricación registradas de la FDA
• Protocolos de cumplimiento de EMA
• Presentaciones regulatorias activas en 3 mercados principales

Fabricación de productos farmacéuticos especializados

Capacidades avanzadas de fabricación biofarmacéutica con tecnologías de producción de proteínas especializadas.

Capacidad de fabricación	Volumen de producción anual	Estándar de control de calidad
Producción de proteínas transgénicas	Hasta 500 kg/año	GMP certificado

Pharming Group N.V. (PHAR) - Modelo de negocio: recursos clave

Tecnologías de ingeniería genética patentada

Pharming Group N.V. utiliza plataformas avanzadas de ingeniería genética diseñadas específicamente para la producción de proteínas en animales transgénicos. La plataforma tecnológica clave de la compañía implica:

• Tecnología de conejo transgénico para la producción de proteínas recombinantes
• Técnicas de modificación genética de precisión
• Procesos de purificación de proteínas patentadas

Categoría de tecnología	Plataforma específica	Estado de desarrollo
Ingeniería genética	Plataforma de conejo transgénico	Totalmente operativo
Producción de proteínas	Extracción de proteínas biofarmacéuticas	Tecnología validada

Equipos de investigación científica especializadas

Pharming mantiene equipos de investigación altamente especializados con experiencia en enfermedades raras y biotecnología.

• Total de personal de investigación: 132 empleados (a partir de 2023)
• Investigadores a nivel de doctorado: 47
• Lugares de investigación: Países Bajos, Estados Unidos

Cartera de propiedades intelectuales

La propiedad intelectual de Pharming representa un recurso clave crítico para la empresa.

Categoría de IP	Número de patentes	Cobertura geográfica
Patentes concedidas	38	Europa, Estados Unidos, internacional
Aplicaciones de patentes pendientes	12	Múltiples jurisdicciones

Instalaciones avanzadas de investigación de biotecnología

Pharming opera instalaciones de investigación y producción de vanguardia.

• Espacio total de la instalación de investigación: 4.500 metros cuadrados
• Nivel de bioseguridad 2 y 3 laboratorios
• Investigación integrada y complejo de fabricación en Leiden, Países Bajos

Experiencia extensa de tratamiento de enfermedades raras

La compañía se enfoca en desarrollar tratamientos para trastornos genéticos raros.

Área terapéutica	Programas de tratamiento actuales	Estadio clínico
Angioedema hereditario	Ruconest (inhibidor recombinante de la esterasa C1)	Aprobado y comercializado
Otras enfermedades raras	Múltiples programas de investigación	Varias etapas de desarrollo

Pharming Group N.V. (PHAR) - Modelo de negocio: propuestas de valor

Tratamientos innovadores para trastornos genéticos raros

Pharming Group N.V. se centra en el desarrollo de terapias para trastornos genéticos raros, específicamente dirigirse:

Trastorno	Producto	Etapa de desarrollo	Potencial de mercado
Angioedema hereditario (Hae)	Ruconesta	Aprobado	Mercado global de $ 500 millones
Enfermedad de Fabry	PRN1006	Fase de ensayo clínico	Mercado potencial de $ 1.2 mil millones

Terapias de reemplazo de enzimas dirigidas

Las terapias de reemplazo de enzimas desarrolladas por farmunda incluyen:

• Reemplazo del inhibidor de la esterasa C1 para HAE
• Terapias de proteínas recombinantes
• Tratamientos enzimáticos con diseñamiento de precisión

Tipo de terapia	Ingresos anuales (2023)	Índice de crecimiento
Ruconesta	€ 111.4 millones	18.4%
Reemplazo de enzimas	€ 132.6 millones	22.7%

Soluciones médicas personalizadas para necesidades de pacientes insatisfechas

Enfoque de Pharming para soluciones médicas personalizadas:

• Tratamientos específicos del trastorno genético
• Desarrollo de medicamentos centrados en el paciente
• Estrategias de medicina de precisión

Intervenciones biotecnológicas avanzadas

Las capacidades biotecnológicas incluyen:

• Plataforma de producción de proteínas patentadas
• Tecnología de proteínas recombinantes
• Investigación de terapia génica

Inversión de I + D	Cantidad de 2023	Porcentaje de ingresos
Gasto de investigación	37,2 millones de euros	28.1%

Productos terapéuticos de alta calidad con eficacia clínica probada

Métricas de rendimiento clínico:

Producto	Tasa de éxito clínico	Satisfacción del paciente
Ruconesta	92.3%	87%
Terapias enzimáticas	89.7%	85%

Pharming Group N.V. (PHAR) - Modelo de negocios: relaciones con los clientes

Médico directo y participación del proveedor de atención médica

Pharming Group N.V. mantiene una participación directa con 782 proveedores de atención médica especializados en 14 países a partir del cuarto trimestre de 2023. El equipo de asuntos médicos de la compañía realiza 213 eventos de educación médica específicas anualmente.

Métrico de compromiso	2023 datos
Proveedores de atención médica totales comprometidos	782
Eventos de educación médica	213
Países cubiertos	14

Programas de apoyo y educación del paciente

Pharming opera programas integrales de apoyo al paciente con 1.647 inscripciones activas de pacientes en 2023.

• Línea de ayuda de apoyo al paciente 24/7
• Portal de recursos de pacientes digitales
• Programas de asistencia financiera

Métrica del programa de pacientes	2023 estadísticas
Inscripciones activas de pacientes	1,647
Apoyo el tiempo de respuesta a la línea de ayuda	<15 minutos

Servicios de consulta médica personalizada

Pharming proporciona Servicios especializados de asesoramiento genético con 437 consultas individuales realizadas en 2023.

Plataformas de información de salud digital

La compañía mantiene una plataforma digital con 92,413 usuarios registrados a diciembre de 2023.

Métrica de plataforma digital	2023 datos
Usuarios registrados	92,413
Usuarios activos mensuales	41,276

Seguimiento clínico en curso y monitoreo

Pharming rastrea a 2,316 pacientes a través de programas de monitoreo clínico a largo plazo en 2023.

• Registros trimestrales del paciente
• Integración de registros de salud electrónicos
• Seguimiento de tratamiento personalizado

Métrica de monitoreo clínico	2023 estadísticas
Total de los pacientes monitoreados	2,316
Frecuencia de monitoreo	Trimestral

Pharming Group N.V. (PHAR) - Modelo de negocio: canales

Ventas directas a centros médicos especializados

Pharming Group N.V. vende directamente sus productos farmacéuticos a centros médicos especializados a través de estrategias de ventas específicas.

Tipo de canal	Número de centros médicos	Cobertura geográfica
Centros de tratamiento de enfermedades raras	127	América del Norte, Europa, Australia
Clínicas especializadas de hemofilia	84	Estados Unidos, Unión Europea

Redes de distribución farmacéutica

Pharming utiliza amplias redes de distribución farmacéutica para llegar a proveedores de atención médica y pacientes.

• Distribuidores farmacéuticos primarios: AmerisourceBergen, Cardinal Health
• Asociaciones internacionales de distribución: 6 principales mayoristas farmacéuticos
• Alcance de distribución global: 18 países

Plataformas de información médica en línea

Canales digitales para la difusión de información médica y comunicación del producto.

Tipo de plataforma	Visitantes únicos mensuales	Confacción de contenido
Pharming Corporate Sitio web	42,500	Información del producto, investigación clínica
Portal profesional médico	23,750	Datos científicos, protocolos de tratamiento

Presentaciones de conferencia médica

Las conferencias científicas sirven como canales críticos para la visibilidad del producto y el intercambio de investigaciones.

• Conferencias anuales a las que asistió: 12
• Tipos de conferencias: enfermedades raras, hematología, trastornos genéticos
• Frecuencia de presentación: 8-10 presentaciones científicas por año

Publicación científica y difusión de investigación

Pharming aprovecha las publicaciones académicas y médicas para comunicar los avances científicos.

Categoría de publicación	Número de publicaciones (2023)	Rango de factores de impacto
Revistas revisadas por pares	14	2.5 - 8.7
Investigación de resúmenes	22	N / A

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

Pacientes con enfermedades raras

Pharming Group N.V. se dirige principalmente a pacientes con trastornos genéticos raros específicos, específicamente:

• Pacientes de angioedema hereditario (HAE): aproximadamente 1 de cada 50,000 personas en todo el mundo
• Pacientes con deficiencia de antitripsina alfa-1: prevalencia global estimada de 1 de 2.500 individuos

Enfermedad rara	Población de pacientes global	Valor de mercado del tratamiento
Angioedema hereditario	10,000-15,000 pacientes diagnosticados en Estados Unidos	$ 2.1 mil millones para 2026
Deficiencia de antitripsina alfa-1	100,000 pacientes diagnosticados en Estados Unidos	$ 1.5 mil millones para 2025

Médicos especializados

Los especialistas médicos objetivo incluyen:

• Inmunólogos
• Genetistas
• Hematólogos
• Pulmonólogos

Centros de tratamiento de trastorno genético

Pharming se dirige centros de tratamiento especializados en todo:

• Estados Unidos: 250 centros de tratamiento de trastorno genético especializados
• Unión Europea: 180 centros de tratamiento de trastorno genético especializados
• Región de Asia-Pacífico: 120 centros de tratamiento de trastorno genético especializados

Proveedores de seguro de salud

Región	Número de proveedores de seguros	Tasa de cobertura de enfermedades raras
Estados Unidos	150 proveedores de seguros principales	72% de cobertura para tratamientos de enfermedades raras
unión Europea	95 proveedores de seguros principales	Cobertura del 68% para tratamientos de enfermedades raras

Instituciones de investigación

Pharming colabora con instituciones de investigación que se centran en las terapias genéticas:

• Estados Unidos: 85 instituciones de investigación
• Unión Europea: 62 instituciones de investigación
• Asociaciones de investigación global: 15 colaboraciones activas

Enfoque de investigación	Número de instituciones	Financiación anual de investigación
Enfermedades raras genéticas	47 instituciones	Financiación anual total de $ 350 millones
Terapias de reemplazo de proteínas	38 instituciones	$ 275 millones en el total de fondos anuales

Pharming Group N.V. (PHAR) - Modelo de negocio: Estructura de costos

Extensas inversiones de investigación y desarrollo

En 2022, Pharming Group N.V. reportó gastos de I + D de € 57.2 millones, lo que representa el 35.4% de los gastos operativos totales.

Año	Gastos de I + D (millones de euros)	Porcentaje de gastos operativos
2022	57.2	35.4%
2021	46.8	33.2%

Gastos de ensayos clínicos

Los costos de ensayos clínicos para el producto clave Ruconest® y los desarrollos de tuberías se estimaron en € 22.5 millones en 2022.

• Ensayos clínicos de fase III para tratamientos de enfermedades raras
• Programas de investigación de enfermedades genéticas en curso
• Gastos continuos de contratación y monitoreo de pacientes

Costos de fabricación y producción

Los gastos de fabricación para 2022 totalizaron aproximadamente 38,6 millones de euros.

Categoría de costos	Cantidad (€ millones)
Costos de materia prima	15.3
Mantenimiento de la instalación de producción	8.7
Control de calidad	6.2
Depreciación del equipo	8.4

Gastos de cumplimiento regulatorio

Los costos de cumplimiento regulatorio para 2022 fueron de aproximadamente 12,4 millones de euros.

• Tarifas de presentación de la FDA y EMA
• Documentación regulatoria en curso
• Sistemas de monitoreo de cumplimiento

Mantenimiento de la propiedad intelectual

Los gastos de propiedad intelectual en 2022 alcanzaron € 5.6 millones.

Categoría de gastos de IP	Cantidad (€ millones)
Presentación de patentes	2.3
Mantenimiento de patentes	1.8
Protección legal	1.5

Pharming Group N.V. (PHAR) - Modelo de negocios: flujos de ingresos

Venta de productos farmacéuticos

En 2022, Pharming Group N.V. reportó ingresos totales de € 224.7 millones, con ingresos primarios generados a partir de ventas de Ruconest® (inhibidor de la esterasa C1).

Producto	Ingresos (2022)	Segmento de mercado
Ruconest®	224.7 millones de euros	Tratamiento hereditario de angioedema

Acuerdos de licencia

Pharming tiene asociaciones estratégicas de licencia que generan fuentes de ingresos adicionales.

• Acuerdo de licencia con Horizon Therapeutics para Ruconest®
• Acuerdo de licencia con CSL Behring por derechos globales

Financiación de la colaboración de investigación

La financiación de colaboración de las asociaciones de investigación contribuyó con 12,5 millones de euros en 2022.

Subvenciones gubernamentales e institucionales

Recibió subvenciones de investigación por un total de aproximadamente 3,2 millones de euros en 2022 para la investigación de enfermedades raras.

Ingresos de regalías de tecnologías patentadas

Ingresos de regalías de tecnologías farmacéuticas patentadas estimadas en € 5.6 millones en 2022.

Flujo de ingresos	Cantidad (2022)
Venta de productos	224.7 millones de euros
Acuerdos de licencia	No revelado
Colaboración de investigación	12.5 millones de euros
Subvenciones del gobierno	3.2 millones de euros
Ingreso de regalías	5,6 millones de euros

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.