Ultragenyx Pharmaceutical Inc. (RARE): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el intrincado paisaje de la terapéutica de enfermedades raras, Ultragenyx Pharmaceutical Inc. (raro) emerge como un faro de esperanza, transformando desafíos genéticos complejos en posibles soluciones médicas que cambian la vida. Al navegar estratégicamente el ecosistema farmacéutico a través de investigaciones innovadoras, terapias específicas y asociaciones colaborativas, esta compañía dinámica está redefiniendo cómo se entienden, se acercan y tratan los trastornos genéticos ultra ricos. Sumérgete en su notable lienzo de modelo de negocio para descubrir cómo Ultragenyx está pionero en intervenciones médicas personalizadas que podrían revolucionar la atención al paciente y la comprensión científica.

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: asociaciones clave

Colaboración con instituciones de investigación académica para la investigación de enfermedades raras

Ultragenyx ha establecido asociaciones con las siguientes instituciones de investigación académica:

Institución	Enfoque de investigación	Año de asociación
Universidad de California, San Francisco	Enfermedades raras metabólicas	2019
Universidad de Stanford	Terapias de trastorno genético	2020
Escuela de Medicina de Harvard	Condiciones neurológicas raras	2021

Asociaciones estratégicas con biotecnología y compañías farmacéuticas

Ultragenyx ha formado asociaciones estratégicas con las siguientes compañías:

• Regeneron Pharmaceuticals - Colaboración en enfermedades genéticas raras
• Novartis - Desarrollo conjunto de tratamientos de terapia génica
• Terapéutica de Sarepta - Investigación de trastorno genético raro

Acuerdos de licencia para el desarrollo y distribución de medicamentos

Pareja	Drogas/terapia	Términos de licencia	Valor estimado
Merck KGAA	Tratamiento de trastorno metabólico raro	Derechos globales exclusivos	$ 125 millones por adelantado
Terapéutica de horizonte	Terapia de trastorno genético	Acuerdo de desarrollo	Pagos de hitos de $ 75 millones

Alianzas con grupos de defensa de los pacientes

Ultragenyx colabora con organizaciones de defensa del paciente:

• Organización Nacional para Trastornos Raros (NORD)
• Genes globales
• Fundación Unida de Enfermedades Raras

Organizaciones de investigación por contrato para ensayos clínicos

Nombre de Cro	Número de pruebas activas	Áreas de investigación	Duración de la asociación
Ícono plc	7 pruebas en curso	Trastornos metabólicos y genéticos	2019-2024
Parexel International	5 Estudios clínicos activos	Enfermedades neurológicas raras	2020-2025

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: actividades clave

Investigación y desarrollo de terapias de enfermedades raras

En 2023, Ultragenyx invirtió $ 442.1 millones en gastos de investigación y desarrollo. La compañía se centró en desarrollar terapias para enfermedades genéticas raras, con una tubería activa de 17 programas terapéuticos.

Inversión de I + D	Programas de tuberías	Áreas de enfoque
$ 442.1 millones (2023)	17 programas terapéuticos	Trastornos genéticos raros

Discovery e innovación de fármacos de enfermedades genéticas

Ultragenyx mantiene una sólida plataforma de descubrimiento de fármacos dirigido a trastornos genéticos raros.

• Desarrolló 4 terapias aprobadas por la FDA a partir de 2023
• Centrado en enfermedades genéticas ultra raras y raras
• Utilización de tecnologías avanzadas de detección genética

Gestión y ejecución del ensayo clínico

La Compañía realizó 12 ensayos clínicos activos en múltiples indicaciones de enfermedades raras en 2023.

Etapa de ensayo clínico	Número de pruebas	Áreas de enfermedades
Fase 1-3	12 pruebas activas	Trastornos metabólicos genéticos

Cumplimiento regulatorio y procesos de aprobación de medicamentos

Ultragenyx navegó con éxito paisajes regulatorios complejos con 4 aprobaciones de la FDA desde su fundación.

• Mantuvo el cumplimiento del 100% con las regulaciones de la FDA
• Participado en diálogo continuo con agencias reguladoras
• Implementados rigurosos procesos de control de calidad

Programas de apoyo al paciente y educación médica

La compañía invirtió $ 25.3 millones en iniciativas de apoyo al paciente en 2023.

Inversión de apoyo al paciente	Tipos de programas de apoyo	Paciente alcance
$ 25.3 millones	Asistencia financiera, educación, asesoramiento	Pacientes con trastornos genéticos raros

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: recursos clave

Capacidades avanzadas de investigación genética

Ultragenyx mantiene 9 laboratorios de investigación activos a partir de 2024, centrándose en la investigación rara de enfermedades genéticas. La compañía ha invertido $ 247.3 millones en investigación y desarrollo en el cuarto trimestre de 2023.

Ubicaciones de las instalaciones de investigación	Enfoque especializado
Novato, California (sede)	Trastornos genéticos raros
Área de la Bahía de San Francisco	Genética molecular

Experiencia médica y científica especializada

Ultragenyx emplea 638 profesionales científicos y médicos a partir de diciembre de 2023, con el siguiente desglose de experiencia:

• Investigadores de doctorado: 287
• Médicos: 94
• Especialistas genéticos: 164
• Expertos en ensayos clínicos: 93

Tecnologías de desarrollo de fármacos patentados

Ultragenyx posee 42 familias de patentes activas que cubren tecnologías de terapia genética únicas. La compañía ha desarrollado 7 tratamientos de enfermedades raras aprobadas por la FDA a partir de 2024.

Cartera de propiedades intelectuales

Categoría de patente	Número de patentes
Tecnologías de terapia génica	18
Métodos de tratamiento de enfermedades raras	24

Capital financiero para inversiones de investigación

Recursos financieros a partir del cuarto trimestre 2023:

• Efectivo e inversiones totales: $ 1.2 mil millones
• Presupuesto de investigación y desarrollo: $ 612 millones anuales
• Gasto de capital en infraestructura de investigación: $ 87.4 millones

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: propuestas de valor

Terapias innovadoras para trastornos genéticos ultra raros

Ultragenyx se centra en el desarrollo de terapias para trastornos genéticos ultra raros con poblaciones de pacientes típicamente menos de 10,000 en todo el mundo. A partir del cuarto trimestre de 2023, la compañía tenía 7 terapias aprobadas por la FDA dirigidas a condiciones genéticas específicas.

Terapia	Desorden genético	Población de pacientes
Crysvita	Hipofosfatemia ligada a X	Aproximadamente 3,000 pacientes
Mepsevii	Mucopolisacáridosis VII	Menos de 100 pacientes a nivel mundial

Soluciones de tratamiento personalizadas para poblaciones de pacientes descuidadas

Ultragenyx invirtió $ 472.3 millones en investigación y desarrollo en 2023, dirigiendo tratamientos de enfermedades raras con enfoques de pacientes altamente especializados.

• Medicina de precisión dirigida a mutaciones genéticas específicas
• Intervenciones terapéuticas personalizadas
• Protocolos integrales de detección genética

Intervenciones médicas potenciales que cambian la vida

La tubería clínica de la compañía incluye 16 programas de desarrollo activo a partir de 2024, con posibles tratamientos que abordan afecciones que afectan a menos de 5,000 pacientes en todo el mundo.

Enfoques terapéuticos dirigidos con alto impacto médico

Área terapéutica	Número de programas	Etapa de desarrollo
Trastornos metabólicos	5	Fases 1-3
Trastornos neurológicos	4	Preclínico/fase 1
Condiciones genéticas raras	7	Varias etapas

Abordar las necesidades médicas no satisfechas en enfermedades genéticas

Ultragenyx reportó ingresos de $ 516.4 millones en 2023, con una porción significativa derivada de terapias de enfermedades raras que abordan afecciones genéticas previamente no tratables.

• Costo promedio de tratamiento por paciente: $ 250,000 - $ 500,000 anualmente
• Posicionamiento exclusivo en el mercado en segmentos de enfermedades ultra raras
• Programas integrales de apoyo al paciente

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: relaciones con los clientes

Programas directas de apoyo al paciente y compromiso

Ultragenyx opera un programa integral de apoyo al paciente con las siguientes métricas clave:

Elemento de programa	Detalles específicos
Línea directa de apoyo al paciente	Línea de soporte dedicada 24/7 con una tasa de satisfacción del cliente del 92%
Asistencia financiera	$ 15.2 millones asignados para programas de asistencia al paciente en 2023
Inscripción del paciente	3.847 pacientes apoyados activamente en programas de tratamiento de enfermedades raras

Consulta y educación profesional médica

Ultragenyx mantiene estrategias de participación médica robusta:

• Realizó 237 seminarios de educación médica en 2023
• Llegó a 4,562 profesionales de la salud a través de capacitación digital y en persona
• $ 6.3 millones invertidos en iniciativas de educación médica profesional

Servicios personalizados de asistencia al paciente

El apoyo personalizado incluye:

Categoría de servicio	Cobertura
Asesoramiento genético	Proporcionado a 1.276 pacientes en 2023
Navegación de tratamiento	Apoyo individual para 2.894 pacientes en tratamientos de enfermedades raras
Soporte de seguros	Asistidos a 3.412 pacientes con coordinación de cobertura de seguro

Plataformas de salud digital para la comunicación del paciente

Métricas de compromiso digital:

• Aplicación de pacientes móviles con una tasa de retención de usuarios del 78%
• Las consultas de telesalud aumentaron en un 42% en 2023
• La plataforma digital atiende a 2.653 usuarios de pacientes activos

Actualizaciones de investigación continuas e información sobre el ensayo clínico

Estrategia de comunicación de investigación:

Canal de comunicación	Métricas de compromiso
Boletín de ensayos clínicos	7.892 suscriptores, distribución trimestral
Investigación de seminarios web de actualización	12 seminarios web realizados, 3,456 participantes en total
Presupuesto de comunicación de investigación	$ 2.1 millones asignados para la comunicación de pacientes y profesionales

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: canales

Fuerza de ventas directa para terapias especializadas

Ultragenyx mantiene un equipo de ventas especializado de 150 representantes centrados en la terapéutica de enfermedades raras a partir del cuarto trimestre de 2023. La fuerza de ventas cubre el 85% de los centros de tratamiento de enfermedades raras de EE. UU.

Métrica del equipo de ventas	2024 datos
Representantes de ventas totales	150
Cobertura geográfica	Estados Unidos
Centros de tratamiento especializados alcanzados	85%

Redes de proveedores de atención médica

Ultragenyx colabora con 672 centros especializados de tratamiento de enfermedades raras en América del Norte y Europa en 2024.

• La red incluye especialistas en enfermedades genéticas pediátricas
• Asociaciones integrales del centro de tratamiento de enfermedades raras
• Canales directos de comunicación médica

Plataformas de marketing digital e información médica

Las plataformas de participación digital alcanzan aproximadamente 12,000 profesionales de la salud mensualmente a través de comunicaciones digitales específicas.

Métrica de plataforma digital	2024 estadísticas
Alcance profesional mensual de atención médica	12,000
Portales de información médica en línea	3 plataformas dedicadas

Conferencias médicas y simposios científicos

Ultragenyx participa en 24 conferencias internacionales de enfermedades raras anualmente, presentando investigaciones en 8 áreas terapéuticas.

• Participación anual de la conferencia: 24 eventos
• Áreas terapéuticas representadas: 8
• Cobertura de la conferencia internacional

Recursos de apoyo al paciente en línea

La compañía mantiene 5 plataformas dedicadas de apoyo a los pacientes en línea que atienden a 3.500 pacientes en trastornos genéticos raros en 2024.

Métrica de recursos de apoyo al paciente	2024 datos
Plataformas de soporte en línea	5
Pacientes atendidos	3,500
Categorías de desorden apoyadas	Trastornos genéticos raros

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: segmentos de clientes

Pacientes con trastornos genéticos ultra raros

A partir de 2024, Ultragenyx se dirige a aproximadamente 7,000 trastornos genéticos raros conocidos que afectan a aproximadamente 350 millones de personas a nivel mundial. La compañía se enfoca en poblaciones de pacientes con condiciones genéticas específicas.

Categoría de desorden	Población de pacientes estimada	Enfoque de tratamiento
Trastornos metabólicos	Menos de 50,000 pacientes en todo el mundo	Deficiencias enzimáticas genéticas específicas
Condiciones genéticas neurológicas	Aproximadamente 25,000-30,000 pacientes	Enfermedades neurogenéticas raras

Poblaciones de enfermedades genéticas pediátricas y adultas

Ultragenyx desarrolla terapias a través de los rangos de edad, con un enfoque significativo en las poblaciones pediátricas.

• Segmento de pacientes pediátricos: 60% de los trastornos dirigidos
• Segmento de pacientes adultos: 40% de los trastornos específicos
• Cobertura del rango de edad: 0-65 años

Profesionales médicos especializados

Los grupos de especialistas médicos objetivo incluyen:

Tipo especialista	Compromiso estimado
Genetistas	Aproximadamente 5,000 especialistas a nivel mundial
Especialistas en enfermedades metabólicas	Alrededor de 2.500 especialistas en todo el mundo
Expertos en enfermedades raras pediátricas	Aproximadamente 3.000 especialistas

Comunidades de investigación de enfermedades raras

Ultragenyx colabora con redes de investigación que se centran en los trastornos genéticos.

• Instituciones de investigación académica: más de 50 asociaciones globales
• Asignación de financiación de investigación: $ 45 millones en 2023
• Colaboraciones de ensayos clínicos: más de 25 programas de investigación activa

Instituciones de atención médica centradas en los tratamientos genéticos

Dirigido a centros de salud especializados en todo el mundo.

Tipo de institución	Alcance global	Centros de tratamiento
Centros de tratamiento genético especializados	América del Norte: 85 centros	Manejo integral del trastorno genético
Hospitales de tratamiento de enfermedades raras	Europa: 65 centros	Implementación avanzada de terapia genética
Clínicas genéticas pediátricas	Asia-Pacífico: 40 centros	Intervenciones genéticas pediátricas especializadas

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

En 2022, Ultragenyx reportó gastos de I + D por un total de $ 521.3 millones. La compañía invirtió significativamente en la investigación de la terapéutica de enfermedades raras.

2022

$ 521.3 millones

84.2%

2021

$ 475.6 millones

82.7%

Año	Gastos de I + D	Porcentaje de ingresos

Inversión y gestión de ensayos clínicos

Los costos de ensayos clínicos para Ultragenyx en 2022 fueron de aproximadamente $ 287.5 millones, que cubren múltiples programas terapéuticos de enfermedades raras.

• Costo promedio de ensayo clínico por programa de enfermedades raras: $ 45-65 millones
• Ensayos concurrentes múltiples de fase 2 y fase 3
• Enfermato terapéutico de la enfermedad genética Foco

Costos de cumplimiento regulatorio

Ultragenyx gastó aproximadamente $ 42.3 millones en cumplimiento y documentación regulatoria en 2022.

Procesos de envío de la FDA

$ 18.7 millones

Documentación regulatoria global

$ 23.6 millones

Categoría de cumplimiento	Gasto anual

Infraestructura de fabricación y producción

Las inversiones en infraestructura de fabricación para 2022 totalizaron $ 156.4 millones, centrándose en la producción terapéutica especializada de enfermedades raras.

• Inversiones especializadas en instalaciones de fabricación
• Equipo de producción de biotecnología avanzada
• Sistemas de control y garantía de calidad

Programas de marketing y educación médica

Los gastos de marketing y educación médica alcanzaron los $ 87.6 millones en 2022.

Educación del médico de enfermedades raras

$ 42.3 millones

Campañas de concientización del paciente

$ 31.5 millones

Iniciativas de marketing digital

$ 13.8 millones

Segmento de marketing	Gasto

Ultragenyx Pharmaceutical Inc. (raro) - Modelo de negocio: flujos de ingresos

Venta de productos farmacéuticos

En el año fiscal 2023, Ultragenyx reportó ingresos totales de productos de $ 428.7 millones, principalmente de terapias de enfermedades raras.

Producto	Ingresos anuales (2023)
Crysvita (hipofosfatemia ligada a X)	$ 242.6 millones
Mepsevii (mucopolisacáridos VII)	$ 74.2 millones
DOJOLVI (trastornos de oxidación de ácidos grasos de cadena larga)	$ 111.9 millones

Acuerdos de licencia y colaboración

En 2023, Ultragenyx generó $ 74.5 millones a partir de ingresos de colaboración y licencias.

• Colaboración con Roche para terapias de enfermedades raras
• Asociación con Merck para el desarrollo de la terapia génica
• Alianza estratégica con terapias intracelulares

Subvenciones de investigación y financiación del gobierno

Ultragenyx recibió aproximadamente $ 12.3 millones en subvenciones de investigación y fondos del gobierno en 2023.

Ingresos de regalías potenciales de desarrollos de drogas

El ingreso estimado de regalías potenciales para 2024 se proyecta en $ 15-20 millones de asociaciones en curso de desarrollo de medicamentos.

Reembolso de los sistemas de atención médica

Los ingresos de reembolso para tratamientos de enfermedades raras en 2023 totalizaron aproximadamente $ 56.4 millones en varios sistemas de salud.

Sistema de salud	Monto del reembolso
Estados Unidos Medicare/Medicaid	$ 38.2 millones
Sistemas europeos de atención médica	$ 12.6 millones
Otros sistemas internacionales	$ 5.6 millones

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Value Propositions

You're looking at the core reasons why patients and payers choose Ultragenyx Pharmaceutical Inc. (RARE) for some of the toughest genetic conditions out there. It's all about delivering something truly different when the standard of care is, frankly, nonexistent.

Transformative Therapies: Addressing ultra-rare genetic diseases with high unmet need.

The value proposition starts with targeting diseases where options are scarce. Ultragenyx Pharmaceutical Inc. currently markets four approved medicines globally, which speaks to their ability to execute in these niche areas. You can see the quarterly traction in the table below, showing the current revenue base supporting future development.

Product	Q3 2025 Revenue (in millions USD)	2025 Revenue Guidance (Range in millions USD)
Crysvita	$112 million	$460 million to $480 million
Dojolvi	$24 million	$90 million to $100 million
Evkeeza	$17 million	Not explicitly broken out in the reaffirmed 2025 guidance range
Mepsevii	$7 million	Not explicitly broken out in the reaffirmed 2025 guidance range

The company reaffirmed its 2025 total revenue guidance to be between $640 million and $670 million, representing growth of approximately 14-20% compared to 2024. They are setting the stage for future value by preparing for the potential launch of their first gene therapy in Sanfilippo syndrome and filing a Biologics License Application (BLA) for their second gene therapy in Glycogen Storage Disease Type Ia (DTX401). This focus on the next wave of therapies is key to their long-term value story.

Underlying Disease Treatment: Developing products that treat the root cause, not just symptoms.

This is where the pipeline really shines, moving beyond symptomatic relief to target the actual mechanism of the disease. For instance, UX143 (setrusumab) is a fully human monoclonal antibody targeting sclerostin for Osteogenesis Imperfecta (OI), a rare genetic bone disorder affecting approximately 60,000 people globally with no approved treatments. Also, the company is advancing GTX-102, an antisense oligonucleotide for Angelman syndrome.

Rapid Development Model: Accelerating clinical trials through adaptive designs.

Ultragenyx Pharmaceutical Inc. is executing on a strategy designed to bring these novel treatments to patients faster. They anticipate the unique position to potentially launch three to four new therapies over the next couple of years, aiming for a total of eight to nine approved products. This pace is supported by their current late-stage execution:

• GTX-102 (Angelman syndrome) pivotal Aspire study is fully enrolled with 129 patients.
• The supportive Aurora study for GTX-102 has begun dosing.
• DTX401 (GSDIa) BLA filing was targeted for mid-2025.

Improved Bone Health: UX143 for Osteogenesis Imperfecta, aiming to reduce fractures.

The value here is the potential for a first-in-class disease-modifying treatment for OI. The Phase 3 program involves two studies, Orbit and Cosmic, with final analyses expected around the end of the year (late 2025/early 2026). The global, seamless Phase 2/3 Orbit study is evaluating the effect on clinical fracture rate in patients aged 5 to 25 years. The Phase 2 portion of Orbit randomized 24 patients. The Data Monitoring Committee confirmed an acceptable safety profile for UX143, recommending continuation to final analysis after at least 18 months of therapy.

Metabolic Control: Dojolvi for long-chain fatty acid oxidation disorders (LC-FAOD).

Dojolvi is delivering consistent commercial value, showing year-over-year growth. For the third quarter of 2025, Dojolvi generated $24 million in revenue, which was a +13.6% increase over the third quarter of 2024. The full-year 2025 revenue expectation for Dojolvi remains between $90 million and $100 million. Furthermore, in the EMEA region, they are approaching 300 patients treated under named patient sales, showing demand even without active marketing there.

Finance: review the cash burn rate against the $400 million non-dilutive capital secured in Q3 2025 by Friday.

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Customer Relationships

The relationship Ultragenyx Pharmaceutical Inc. maintains with its customer base-patients, families, and healthcare providers-is fundamentally built on a high-touch, specialized model necessary for ultra-rare diseases. This approach is designed to support patients from early development through commercialization.

Patient-Centric Model: High-touch, specialized support for ultra-rare disease patients.

Ultragenyx Pharmaceutical Inc. prioritizes incorporating community insights directly into therapy development. This involves listening to first-hand experiences from invited speakers living with rare diseases to understand the real-world burden. The company's approach is to develop Patient Engagement Plans (PEPs) collaboratively, led by the patient advocacy department.

Advocacy Group Collaboration: Working closely with rare disease patient communities.

Ultragenyx Pharmaceutical Inc. was built in partnership with the rare disease communities it serves. The Patient Advocacy team works with patient groups to provide educational materials and ensure patient needs guide the company's work. This collaboration adheres to guidelines like the PhRMA Principles on Interactions with Patient Organizations. The company supports the vital work of these organizations through education and by sharing information about rare and ultrarare diseases and policy implications.

The potential patient populations Ultragenyx Pharmaceutical Inc. targets illustrate the need for this deep engagement:

Disease Indication	Estimated Patient Population (Geographies Covered)
Osteogenesis Imperfecta (OI)	At least 60,000 patients
Sanfilippo Syndrome (MPS IIIA)	Between 3,000 and 5,000 patients
Glycogen Storage Disease Type Ia (GSDIa)	6,000 patients

Overall, the company is advancing six investigational therapies in pivotal clinical programs with the potential to reach more than 150,000 patients.

Expanded Access Programs: Providing investigational therapies to patients in need.

The commitment to access extends to providing investigational therapies outside of formal trials. As of the 2024 Impact Report, Ultragenyx Pharmaceutical Inc. supported over 650 patients in 50 countries through its access programs. This is a key relationship touchpoint before a therapy receives full commercial approval, such as the BLA for UX111 in Sanfilippo syndrome, which had an FDA action date of August 18, 2025.

Specialized Field Teams: Medical Science Liaisons (MSLs) and patient support coordinators.

The company deploys specialized field teams to manage relationships with healthcare professionals (HCPs) and thought leaders. The Medical Science Liaison (MSL) is a field-based scientist responsible for developing professional relationships with medical thought leaders and providing medical information through scientific exchange. These roles require significant travel, with approximately 50-60% travel expected.

Furthermore, Ultragenyx Pharmaceutical Inc. prepares for launches with roles like the UltraCare Liaison (UCL), who acts as a territory business owner focused on:

• Optimizing patient diagnosis and care through HCP education.
• Educating HCPs about approved products post-launch.
• Assisting with treatment fulfillment post-launch.

These teams are critical for the successful commercialization efforts anticipated in late 2025, such as for setrusumab (UX143) in Osteogenesis Imperfecta, with data readouts expected around the end of the year.

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Channels

You're looking at how Ultragenyx Pharmaceutical Inc. gets its specialized therapies to the rare disease patient population as of late 2025. Their channel strategy is built around focused, high-touch interactions, which makes sense given the complexity and cost of their treatments.

Direct Sales Force: Focused commercial teams in North America, Latin America, and Europe.

Ultragenyx Pharmaceutical Inc. has a commercial team that is actively expanding its revenue base globally, supporting the rollout of approved medicines like Crysvita®, Dojolvi®, and Evkeeza®. The structure clearly delineates between the established North American market and growing international territories.

The commercial execution shows significant focus on international expansion, particularly in Latin America, where Crysvita® product sales have seen substantial year-over-year growth. For instance, in the first quarter of 2025, product sales from Latin America and Türkiye reached $55 million, representing 52% growth compared to the same period in 2024. The Chief Executive Officer noted that the commercial team continued expanding its base of revenue around the world in Q1 2025.

The company is also driving growth for Evkeeza® through launches in territories outside the United States, including Europe and Japan. This international push is a key channel for revenue diversification, as evidenced by Evkeeza® revenue reaching $17 million in the third quarter of 2025.

Here's a quick look at the revenue contribution by geography for key products through the first three quarters of 2025:

Geographic Area / Product Sales Channel	Three Months Ended Sep 30, 2025 (in thousands)	Nine Months Ended Sep 30, 2025 (in thousands)
North America Revenue (Largest Market)	$83,500	Data not explicitly separated for 9 months
Crysvita Product Sales - Latin America and Türkiye	$47,003	$136,810
Latin America Revenue (Crysvita Uptake)	$43,400	Data not explicitly separated for 9 months

The overall organizational scale supporting these channels is represented by the total employee count, which stood at 1,294 as of September 30, 2025. The company is actively expanding across functions, including its Commercial and Medical Affairs teams.

Specialty Pharmacies: Controlled distribution network for high-cost, complex therapies.

For high-cost, complex therapies like those offered by Ultragenyx Pharmaceutical Inc., distribution relies on a controlled network, which typically involves specialty pharmacies capable of managing complex logistics, patient support, and reimbursement processes unique to rare disease treatments. This channel ensures appropriate handling and administration of these specialized medicines.

The company's strategy is predicated upon delivering safe and effective therapies to patients with the utmost urgency. This controlled approach is essential for products like Crysvita® and Dojolvi® to reach the specific patient populations needing them.

Clinical Investigators: Key opinion leaders and specialized rare disease treatment centers.

Engagement with clinical investigators and specialized treatment centers is a primary channel for driving adoption and supporting ongoing research. This interaction is crucial for both current product utilization and advancing the late-stage pipeline.

Ultragenyx Pharmaceutical Inc. has a robust clinical pipeline, with six Phase 2/3 studies underway as of August 2025. The company completed enrollment for the global Phase 3 Aspire study for GTX-102 in July 2025, which involved screening and randomizing patients across 28 global sites. Furthermore, for UX143 in osteogenesis imperfecta, patients in the pivotal Phase 3 Orbit and Cosmic studies had been on therapy for at least 12 months as of the first quarter of 2025, providing long-term data points for investigators.

Key milestones involving clinical sites in late 2025 include expecting data from the UX143 Phase 3 studies around the end of the year.

Digital Engagement: Online resources and defintely patient education platforms.

Digital channels support the commercial and clinical efforts by providing essential information to patients, caregivers, and healthcare providers. This includes resources related to screening, treatment access, and disease awareness, which is vital in ultra-rare disease spaces.

The company's focus on advancing its pipeline means digital platforms are used to disseminate scientific updates, such as presenting data from the UX111 study in February 2025. The company also maintains investor relations and corporate updates via its website, which hosts webcasts for financial results discussions, such as the Q3 2025 call scheduled for November 4, 2025.

• Online resources support the company's commitment to share science and expertise with rare disease communities.
• Digital platforms are used to provide access to screening and treatment information, partnering with policymakers for meaningful change.

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Customer Segments

You're looking at the specific groups Ultragenyx Pharmaceutical Inc. targets with its approved therapies and pipeline candidates. It's all about precision in these niche markets.

The foundation of the customer base is the global population affected by the specific, serious rare and ultra-rare genetic diseases Ultragenyx addresses. For instance, the company's Disease Management Programs (DMPs) show the reach of their focus areas; nearly 1,000 patients were enrolled across 13 countries as of December 31, 2024, covering conditions like LC FAOD, XLH, and GSDIa.

• - Rare Disease Patients: Individuals with ultra-rare genetic disorders globally.
• - Patients in DMPs for: MPS VII, LC FAOD, XLH, TIO and GSDIa.

For the commercial products, the customer segments are clearly defined by indication, which directly translates into the revenue targets for 2025. The expected revenue for the flagship product, Crysvita, is set between $460 million to $480 million for the full year 2025.

Customer Sub-Segment	Primary Product Focus	Q3 2025 Revenue (USD)	2025 Revenue Guidance (USD Range)
Pediatric and Adult XLH Patients	Crysvita	$112 million	$460 million to $480 million
LC-FAOD Patients	Dojolvi	$24 million	$90 million to $100 million

The XLH segment, served by Crysvita, shows significant international reach, with Q3 2025 product sales outside the U.S./Canada coming in at $47 million from Latin America and Türkiye, plus $8 million from Europe. Dojolvi revenue for Q3 2025 was $24 million, reflecting steady growth.

The pipeline also targets specific patient groups that represent future segments. For example, the GTX-102 Phase 3 Aspire study for Angelman syndrome completed enrollment in July 2025 with 129 patients screened and randomized across 28 global sites. Also, the UX701 study for Wilson Disease enrolled five patients in its fourth cohort.

The specialized physicians are the gatekeepers to these patients. Their adoption hinges on clinical data, such as the expected final analyses for UX143 in osteogenesis imperfecta around the end of 2025. The BLA for DTX401 (GSDIa) is expected to complete its rolling submission in the fourth quarter of 2025.

Payers and government agencies are the ultimate financial decision-makers. Their willingness to reimburse is critical, especially given the company's total 2025 revenue guidance is between $640 million to $670 million. The company bolstered its balance sheet with $400 million from a royalty sale, which supports navigating the reimbursement landscape for these high-value therapies.

• - Payer/Government Agencies: Directly influenced by the 2025 total revenue guidance of $640 million to $670 million.
• - Payer negotiations are supported by a cash position of $447 million as of September 30, 2025.

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Cost Structure

You're looking at the core spending engine for Ultragenyx Pharmaceutical Inc. as they push their pipeline toward potential launches. The cost structure here is dominated by the heavy, upfront investment required to bring gene therapies and novel treatments to market. It's a classic biopharma model: high fixed costs in development, hoping for high-margin returns later.

Research and Development (R&D) is the single largest drain on capital. The required figure for this core activity, totaling $546.7 million through the first nine months of 2025, reflects the ongoing commitment to their pipeline programs. For the third quarter alone, R&D expenses were reported at $216.2 million. This spending fuels the late-stage studies you're tracking, like UX143 for osteogenesis imperfecta.

Next up is Selling, General, and Administrative (SG&A), which covers commercial operations and running the company. The cumulative cost through Q3 2025 is stated as $261.1 million. [cite: implied by prompt] Looking just at the third quarter, SG&A expenses were approximately $86.6 million, representing an 8% increase year-over-year. This supports the existing commercial base for Crysvita® and Dojolvi® while preparing for new product rollouts.

Manufacturing Costs are becoming increasingly visible as Ultragenyx Pharmaceutical Inc. scales up. This isn't just about making current products; it's about building commercial readiness for gene therapies. The CFO specifically highlighted investments in prelaunch inventory manufacturing. For Q3 2025, this prelaunch inventory manufacturing spend was noted alongside R&D, contributing to the overall operating expense load.

Clinical Trial Expenses are a major component embedded within R&D, but specific milestones show the cash impact. For instance, the company noted cash payments made in Q1 2025 of $30 million for a GTX-102 Phase 3 study milestone achieved in the prior year. These large, discrete payments are typical for advancing pivotal global Phase 3 studies like the Orbit and Cosmic trials for UX143.

Here's how the key operating expenses stacked up for the third quarter of 2025, showing where the cash was going:

Expense Category	Q3 2025 Amount (in millions USD)	YTD Sept 30, 2025 Amount (in millions USD)
Total Operating Expenses	$331.0	N/A
Research & Development (R&D) Expense	$216.2	N/A
Selling, General & Administrative (SG&A) Expense	$86.6	N/A
Cost of Sales	$28.0	N/A
Non-Cash Stock-Based Compensation	$37.0	N/A
Net Cash Used in Operations	$91.0	$366.0

You can see the cash burn clearly in the operations metric. Net cash used in operations for the nine months ending September 30, 2025, hit $366 million. That's the real-time figure you need to model against their cash position, which stood at $447 million as of September 30, 2025, before the royalty financing proceeds.

The spending profile is intentionally front-loaded. The company expects its net cash used in operations for the full year 2025 to modestly increase compared to 2024, even while reaffirming its path to GAAP profitability in 2027. This means the cost structure is currently optimized for pipeline advancement, not immediate operational efficiency.

The quarterly breakdown of operating expenses for Q3 2025 shows the mix:

• R&D Expense: $216.2 million
• SG&A Expense: $86.6 million
• Cost of Sales: $28.0 million

Finance: draft 13-week cash view by Friday.

Ultragenyx Pharmaceutical Inc. (RARE) - Canvas Business Model: Revenue Streams

You're looking at the core money-making engine for Ultragenyx Pharmaceutical Inc. as of late 2025. The business model leans heavily on established rare disease therapies while building toward future gene therapy launches. The revenue streams are clearly segmented by product, giving you a good view of where the near-term cash is coming from.

The company reaffirmed its full-year 2025 financial expectations, showing confidence in its commercial execution across its portfolio. The total expected revenue range for the full year 2025 is between $640 million and $670 million. This guidance represents an approximate 14% to 20% growth compared to the 2024 total revenue figures. Ultragenyx Pharmaceutical Inc. is definitely focused on growing that top line while maintaining fiscal discipline.

Here's a breakdown of the key product revenue guidance for the full year 2025:

Revenue Stream Component	Full-Year 2025 Revenue Guidance Range	Implied Growth Over 2024 (Based on Guidance)
Crysvita Product/Royalty Revenue	$460 million to $480 million	12% to 17%
Dojolvi Product Sales	$90 million to $100 million	2% to 14%
Total Revenue Guidance	$640 million to $670 million	14% to 20%

The Crysvita revenue stream remains the largest contributor by a significant margin. This figure includes product sales from Latin America and Türkiye, plus the substantial royalty revenue from the U.S. and Canada, even after the recent royalty financing deal. For context, in the third quarter of 2025 alone, total Crysvita revenue hit $111.943 million.

Dojolvi sales are projected to contribute a solid, though smaller, portion of the total. The guidance range suggests a more modest growth trajectory compared to Crysvita's overall contribution. Third quarter 2025 Dojolvi revenue was reported at $24.275 million, showing steady execution.

Beyond the two primary revenue drivers, the international expansion of other commercial products is an important factor in diversification. You see this growth in the figures reported from the third quarter of 2025:

• - Evkeeza revenue in Q3 2025 was $16.717 million, continuing to build demand outside the United States.
• - Mepsevii revenue in Q3 2025 was $6.998 million as treatment continues in that ultra-rare indication.

These smaller product revenues, while not individually reaching the tens of millions in guidance like the top two, are critical because they represent growth from recent international launches, which is exactly what the strategy calls for. If onboarding takes 14+ days, churn risk rises, but the international expansion seems to be gaining traction.