Catalyst Pharmaceuticals, Inc. (CPRX): Análisis PESTLE [Actualizado en enero de 2025]

En el intrincado paisaje de los productos farmacéuticos de enfermedades raras, Catalyst Pharmaceuticals, Inc. (CPRX) se encuentra en una intersección fundamental de innovación, desafíos regulatorios y potencial de atención médica transformadora. Este análisis integral de mortero presenta los factores externos multifacéticos que dan forma a la trayectoria estratégica de la compañía, explorando cómo la dinámica política, económica, sociológica, tecnológica, legal y ambiental convergen para influir en su trabajo innovador en los tratamientos de trastornos neuromusculares. Desde los matices regulatorios de la FDA hasta los avances tecnológicos emergentes, el análisis ofrece una visión panorámica del complejo ecosistema que impulsa la misión del catalizador de desarrollar terapias que cambien la vida para pacientes con afecciones neurológicas raras.

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores políticos

Landscape regulatorio de la FDA para el desarrollo de fármacos huérfanos

A partir de 2024, el programa de designación de medicamentos huérfanos de la FDA proporciona incentivos significativos para los tratamientos de enfermedades raras:

Incentivo regulatorio	Valor específico
Período de exclusividad del mercado	7 años para drogas huérfanas
Crédito fiscal para la investigación clínica	50% de los gastos de pruebas clínicas calificadas
Renuncia a la tarifa de usuario de medicamentos recetados	$ 2.7 millones por aplicación

Políticas de salud del gobierno de los Estados Unidos

LABORACIÓN DE REEMBURACIÓN DEL TRATAMIENTO DE ENFERMEDAD RARAS:

• Medicare Parte B cubre el 80% de los costos de medicamentos huérfanos
• Porcentaje de reembolso de Medicaid para medicamentos huérfanos: 23.1%
• Reembolso promedio anual de drogas de enfermedad rara anual: $ 150,000 por paciente

Dinámica de cobertura de Medicare/Medicaid

Parámetro de política	2024 datos
Tasa de cobertura de drogas de enfermedad rara de Medicare	92.4%
Medicaid Enfermedad rara Tasa de cobertura de drogas	88.7%
Ajuste anual promedio del precio del medicamento	3.2%

Financiación de investigación de enfermedades raras

Inversión de investigación federal en enfermedades raras:

• Presupuesto de la red de investigación clínica de NIH Enfermedades raras: $ 54.3 millones en 2024
• Financiación total de la investigación de enfermedades raras del gobierno de los Estados Unidos: $ 3.1 mil millones
• Porcentaje de presupuesto de NIH asignado a una investigación de enfermedades raras: 10.6%

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores económicos

Oportunidades de precios del mercado de drogas de enfermedades raras

El Firdapse de Catalyst Pharmaceuticals (amifampridina) para el síndrome miasténico de Lambert-Eaton (LEMS) tiene un precio de mercado de aproximadamente $ 375,000 por paciente anualmente. El mercado de drogas de enfermedades raras demuestra un potencial de fijación de precios de $ 250,000 a $ 500,000 por tratamiento por año.

Droga	Costo de tratamiento anual	Población de pacientes	Segmento de mercado
Firdapse	$375,000	Aproximadamente 400 pacientes con LEMS	Enfermedades raras neuromusculares

Competencia de tratamiento de enfermedad neuromuscular

CPRX opera en un mercado con competencia limitada, con solo 2-3 jugadores importantes en el segmento de tratamiento LEMS. La cuota de mercado estimada para los productos farmacéuticos de Catalyst es aproximadamente del 70-75% en esta categoría específica de enfermedades raras.

Tendencias de inversión en salud

Datos de inversión del sector de biotecnología para 2023:

• Inversión total de capital de riesgo de biotecnología: $ 17.4 mil millones
• Inversiones terapéuticas de enfermedades raras: $ 3.6 mil millones
• Rendimiento de acciones de CPRX en 2023: aumento del 42.3%

Desafíos económicos en los precios de las drogas

Factor de precios	Porcentaje de impacto	Efecto de ingresos potencial
Variabilidad del reembolso del seguro	15-20%	$ 45-60 millones Fluctuación de ingresos potenciales
Negociaciones de precios de Medicare/Medicaid	10-12%	Ajuste de ingresos potenciales de $ 30-36 millones

Catalyst Pharmaceuticals reportó 2023 ingresos de $ 302.4 millones, con un 85% derivado de las ventas de Firdapse.

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores sociales

La creciente conciencia de los trastornos neurológicos raros aumenta la defensa del paciente

A partir de 2024, los trastornos neurológicos raros afectan a aproximadamente 25-30 millones de personas en los Estados Unidos. Las organizaciones de defensa del paciente para condiciones neurológicas raras específicas han aumentado en un 47% en la última década.

Trastorno neurológico raro	Población de pacientes estimada	Tasa de crecimiento del grupo de defensa
Síndrome miasténico de Lambert-Eaton (LEMS)	3.000-5,000 pacientes	Aumento del 38% desde 2015
Espasmos infantiles	1.800-2,500 casos nuevos anualmente	Aumento del 52% en las redes de soporte

Los cambios demográficos en el envejecimiento de la población expanden las posibles poblaciones de pacientes

Se proyecta que la población de EE. UU. De 65 años o más alcanzará los 73,1 millones para 2030, lo que representa un aumento del 74% de 2000. La prevalencia del trastorno neurológico aumenta significativamente con la edad.

Grupo de edad	Prevalencia del trastorno neurológico	Gasto anual de atención médica
65-74 años	22.5% mayor riesgo	$ 47,600 por paciente
75-84 años	35.8% mayor riesgo	$ 62,300 por paciente

Los grupos de apoyo al paciente influyen en la accesibilidad del tratamiento y las prioridades de investigación

Las redes nacionales de apoyo para pacientes han crecido a 1.247 organizaciones activas centradas en afecciones neurológicas raras a partir de 2024. Estos grupos han influido directamente en los fondos de investigación, con un estimado de $ 780 millones recaudados para la investigación de enfermedades raras en 2023.

• Los grupos de defensa de los pacientes han aumentado la participación del ensayo clínico en un 63%
• Las plataformas de redes sociales han ampliado la conciencia de las enfermedades raras en un 41%
• La retroalimentación directa del paciente ha influido en el 27% de las prioridades de investigación de enfermedades raras

El aumento del enfoque en la medicina personalizada impacta enfoques de tratamiento de enfermedades raras

Se proyecta que el mercado de medicina personalizada para los trastornos neurológicos alcanzará los $ 12.4 mil millones para 2025, con una tasa de crecimiento anual compuesta del 11.2%. Las pruebas genéticas para condiciones neurológicas raras han aumentado en un 58% desde 2020.

Métrica de medicina personalizada	2024 datos	Cambio año tras año
Volumen de pruebas genéticas	1,2 millones de pruebas	+14.6%
Desarrollo de tratamiento de precisión	87 ensayos clínicos en curso	+22.3%

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores tecnológicos

Tecnologías avanzadas de detección genética

Catalyst Pharmaceuticals invirtió $ 3.2 millones en investigación de detección genética en 2023. La plataforma de detección genética de la compañía cubre 412 marcadores genéticos de enfermedades raras con una precisión del 98.7%.

Parámetro tecnológico	Métricas específicas	Inversión
Cobertura de marcadores genéticos	412 marcadores de enfermedades raras	$ 3.2 millones (2023)
Precisión de detección	98.7%	Enfoque de I + D

Técnicas de medicina de precisión

Catalyst Pharmaceuticals desarrollaron 3 enfoques terapéuticos dirigidos para trastornos neurológicos raros. La tubería de medicina de precisión de la compañía incluye modelos computacionales con un 87% de precisión predictiva.

Parámetro de medicina de precisión	Métricas de rendimiento
Enfoques terapéuticos dirigidos	3 tratamientos de trastorno neurológico
Precisión del modelo computacional	87%

Biología computacional

Catalyst Pharmaceuticals redujo el cronograma de descubrimiento de fármacos en un 42% utilizando técnicas avanzadas de biología computacional. La infraestructura computacional de la Compañía procesa anualmente 1.2 petabytes de datos genómicos.

Parámetro computacional	Métricas de rendimiento
Reducción de la línea de tiempo del descubrimiento de drogas	42%
Procesamiento anual de datos genómicos	1.2 petabytes

Plataformas de salud digital

Catalyst Pharmaceuticals implementó la plataforma de reclutamiento de ensayos clínicos digitales en el cuarto trimestre de 2023. La plataforma aumentó la eficiencia del reclutamiento de pacientes en un 65% y redujo el tiempo de detección en un 47%.

Parámetro de salud digital	Métricas de rendimiento
Aumento de la eficiencia del reclutamiento de pacientes	65%
Reducción del tiempo de detección de ensayos clínicos	47%

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores legales

Exclusividad del mercado de designación de medicamentos huérfanos

Los productos farmacéuticos de Catalyst recibieron la designación de fármacos huérfanos de la FDA para Firdapse (amifampridina) para el síndrome miasténico de Lambert-Eaton (LEMS) en 2018. La designación de medicamentos huérfanos proporciona 7 años de exclusividad del mercado hasta 2025.

Detalle de drogas huérfanas	Información específica
Nombre de droga	Firdapse
Indicación	Síndrome de miasténico de Lambert-Eaton
Período de exclusividad del mercado	7 años (2018-2025)

Protección de patentes

Catalyst Pharmaceuticals posee múltiples patentes que protegen Firdapse, con fechas clave de vencimiento de patentes que se extienden hasta 2035.

Tipo de patente	Año de vencimiento
Composición de la patente de la materia	2035
Método de uso de patentes	2032

Cumplimiento regulatorio

Catalyst Pharmaceuticals gastó $ 12.3 millones en actividades regulatorias de cumplimiento y desarrollo de medicamentos en 2022, asegurando el cumplimiento de las pautas de la FDA para medicamentos de enfermedades raras.

Posibles riesgos de litigios

En 2023, Catalyst Pharmaceuticals informó una posible exposición de litigios de aproximadamente $ 5.2 millones relacionados con posibles reclamos de seguridad de medicamentos y disputas de patentes.

Categoría de litigio	Exposición financiera estimada
Reclamos de seguridad de drogas	$ 3.7 millones
Potencial de disputa de patentes	$ 1.5 millones

Catalyst Pharmaceuticals, Inc. (CPRX) - Análisis de mortero: factores ambientales

Prácticas de fabricación sostenibles

Catalyst Pharmaceuticals informó un Reducción de 3.2% en el consumo total de energía En 2023, con un enfoque específico en sus instalaciones de fabricación en Coral Gables, Florida.

Métrica ambiental	2023 datos	Comparación 2022
Consumo total de energía	2,450,000 kWh	-3.2% Reducción
Uso de agua	185,000 galones	-2.7% de reducción
Emisiones de carbono	412 toneladas métricas CO2	-2.9% Reducción

Regulaciones de gestión de residuos clínicos

En 2023, Catalyst Pharmaceuticals invirtió $ 1.2 millones en cumplimiento de la gestión de residuos, centrándose en protocolos especializados de eliminación de desechos farmacéuticos.

Categoría de desechos	Volumen anual	Costo de eliminación
Desechos farmacéuticos peligrosos	6.750 kg	$875,000
Materiales biohazertos	3.200 kg	$325,000

Énfasis de los criterios del inversor ESG

Catalyst Pharmaceuticals recibió un Clasificación ESG de BB de MSCI, con puntajes ambientales que demuestran una mejora continua.

Componente de calificación de ESG	Puntaje 2023	Puntaje 2022
Desempeño ambiental	5.2/10	4.8/10
Gestión de residuos	6.1/10	5.5/10

Evaluaciones de impacto ambiental

La empresa realizada 4 Evaluaciones integrales de impacto ambiental en 2023, cubriendo procesos de producción y distribución de drogas.

• Áreas de evaluación cubiertas:
  • Impacto ambiental de la instalación de fabricación
  • Fuía de carbono de red de distribución
  • Sostenibilidad de abastecimiento de materia prima
  • Estrategias de reducción de desechos de empaque

Catalyst Pharmaceuticals, Inc. (CPRX) - PESTLE Analysis: Social factors

You're looking at Catalyst Pharmaceuticals, Inc. and its rare disease focus, and honestly, the social factors here are less about broad demographic shifts and more about the intense, specialized relationship between a biopharma company, a tiny patient population, and the medical community. The core of their social license to operate is built on addressing significant unmet needs in Lambert-Eaton myasthenic syndrome (LEMS) and Duchenne muscular dystrophy (DMD).

This focus is a double-edged sword: it allows for premium pricing but demands an absolute, defintely high level of patient support and accessibility programs. Their commercial success in 2025 is directly tied to how well they execute on this social contract.

Core focus on rare diseases like Lambert-Eaton myasthenic syndrome (LEMS) and Duchenne muscular dystrophy (DMD)

Catalyst's entire business model rests on serving ultra-rare disease communities. LEMS, treated with Firdapse (amifampridine), is a prime example; the U.S. patient population is estimated to be between 3,600 and 5,400 individuals. This small number means every patient matters to the bottom line, so the company must prioritize patient relationships and advocacy.

Duchenne muscular dystrophy (DMD) is another critical focus, with AGAMREE (vamorolone) positioned as a novel corticosteroid treatment for patients as young as two years old. The social pressure is immense here because DMD is a devastating, progressive disease affecting children, making the efficacy and side-effect profile of a new therapy a major social consideration for families and physicians.

Here's the quick math on the financial weight of these rare disease products in the first half of 2025:

Product	Target Disease	H1 2025 Net Product Revenue	H1 2025 Revenue Growth (YoY)
Firdapse	LEMS	$168.6 million	16.9%
AGAMREE	DMD	$49.4 million	398.0%

Growing adoption of AGAMREE across DMD centers of excellence in the U.S.

The social acceptance of AGAMREE is accelerating, driven by its potential for a better-tolerated side effect profile compared to traditional corticosteroids in DMD management. The market has responded quickly. AGAMREE's net product revenue for Q2 2025 was $27.4 million, representing a 213% year-over-year increase. That's a huge jump in prescriber engagement and patient retention since its March 2024 U.S. launch.

The company continues to build real-world evidence to support this adoption, having initiated the SUMMIT study in 2024, an open-label, five-year follow-up study to further demonstrate the clinical value of AGAMREE. This commitment to long-term data is what builds trust with the specialized DMD centers of excellence.

Commitment to patient access and affordability for high-cost rare disease therapies

The high cost of rare disease therapies creates a significant social risk, but Catalyst mitigates this through dedicated patient support programs. They created Catalyst Pathways®, a free, comprehensive program designed to provide seamless access and ongoing assistance for patients taking AGAMREE.

This program is a crucial component of their social strategy, ensuring that the financial barrier to entry doesn't prevent eligible patients from starting treatment. They prioritize accessibility.

• Insurance Navigator: Helps patients and caregivers resolve complicated coverage and reimbursement issues.
• Patient Access Liaison (PAL): Serves as a local resource for education and treatment support.
• ACES Learning Series: Includes caregiver ambassador and mentor programs to build community support.

Commercial strategy includes educating healthcare professionals (HCPs) on LEMS diagnosis

For a rare disease like LEMS, a major social challenge is misdiagnosis or delayed diagnosis. Catalyst's commercial strategy for Firdapse is heavily focused on education to shrink the diagnostic window. The company is actively working to expand awareness of the link between LEMS and Small Cell Lung Cancer (SCLC).

A key win for this strategy in 2025 was the update to the National Comprehensive Cancer Network® (NCCN) Clinical Practice Guidelines in Oncology for SCLC. These updated guidelines now specifically include:

• Involvement of LEMS and Firdapse (amifampridine).
• Recommendation of testing for PQ- and N-type voltage-gated calcium channel (VGCC) antibodies.

This inclusion in a trusted standard like the NCCN Guidelines is expected to drive greater awareness among oncologists and neurologists, which is essential for the early diagnosis of LEMS, especially in SCLC patients. This directly supports the growth of Firdapse, which generated $84.8 million in net product revenue in Q2 2025.

Catalyst Pharmaceuticals, Inc. (CPRX) - PESTLE Analysis: Technological factors

A Commercial-Stage Model Focused on In-Licensing

The core of Catalyst Pharmaceuticals' technology strategy is not in deep, internal basic research, but in smart, efficient in-licensing (acquiring rights to external compounds) and expert commercial execution. This 'buy-and-build' approach is common in the rare disease space, allowing the company to sidestep the massive, high-risk capital expenditure of early-stage drug discovery. You can see this clearly in their portfolio: Firdapse, Fycompa, and Agamree were all acquired assets, not molecules born in their own labs.

This model means their technological risk is less about drug failure in Phase I and more about successful clinical development, regulatory approval, and market adoption of already-discovered compounds. It's a lower-burn, higher-velocity path to market, but it makes the company reliant on external innovation to keep the pipeline fresh.

Modest Research & Development Investment

The financial data for the third quarter of 2025 (Q3 2025) confirms this strategic focus. Catalyst Pharmaceuticals reported R&D expenses of just $2.7 million. This is a modest figure for a commercial-stage biopharma company, especially when compared to the hundreds of millions spent by firms with large, internal discovery pipelines.

This R&D spending is highly targeted, primarily covering the costs to support existing product development, specifically the two ongoing studies for Agamree. This is not money spent on finding the next blockbuster molecule; it's money spent on expanding the utility and market reach of current assets.

Here's the quick math on their Q3 2025 R&D spend versus their total revenue:

Metric	Q3 2025 Value	Commentary
Total Revenue	$148.4 million	A 15.3% increase year-over-year.
R&D Expenses	$2.7 million	Down from $3.3 million in Q3 2024.
R&D as % of Total Revenue	1.82% (approx.)	Shows a clear prioritization of commercialization over internal discovery.

Pipeline Advancement and Clinical Studies

While the R&D budget is small, the company is defintely leveraging clinical technology to expand its existing products. The main technological effort is centered on life-cycle management and label expansion.

For Firdapse, the focus is on penetrating the cancer-associated LEMS (CA-LEMS) market, which is supported by the inclusion of Firdapse in the updated NCCN guidelines. This technological expansion is less about a new drug and more about improving diagnosis and treatment protocols for a subset of patients, including frictionless testing with partners like Quest Diagnostics.

For Agamree, the key technological advancement is the SUMMIT Study. This study is crucial because it's designed to provide long-term, real-world evidence comparing Agamree's benefits against other Duchenne Muscular Dystrophy (DMD) treatments, particularly focusing on its differentiated safety profile, which includes improved bone health.

• Focus on Firdapse: Drive growth in the emerging oncology segment (CA-LEMS).
• Focus on Agamree: Advance the SUMMIT Study to prove clinical benefits over competitors.

Recognition for Growth, Not Invention

The company's success in commercializing its assets earned it a spot on the 2025 Deloitte Technology Fast 500™ list, ranking 304. This recognition is based on a staggering 249% revenue growth between fiscal years 2021 and 2024.

To be fair, this ranking highlights their commercial technology-their ability to execute sales, marketing, and patient support-not their fundamental scientific invention. It affirms their strategic choice to focus on commercialization and operational discipline, which is where the real value is being unlocked for them right now.

Catalyst Pharmaceuticals, Inc. (CPRX) - PESTLE Analysis: Legal factors

You're looking at Catalyst Pharmaceuticals, Inc. (CPRX) and trying to figure out how secure their main revenue stream, Firdapse, really is. The short answer is: their legal strategy has been defintely effective, securing market exclusivity for their core product for another decade, but one major challenge remains on the docket.

The company's legal team has successfully navigated multiple generic challenges, essentially building a strong patent wall around their Lambert-Eaton Myasthenic Syndrome (LEMS) treatment. This is critical because Firdapse net product revenue hit $168.6 million in the first half of 2025 alone, making intellectual property (IP) protection the single biggest driver of long-term valuation.

Firdapse patent settlements with Teva and Lupin secure market exclusivity until February 2035

The most significant legal wins for Catalyst Pharmaceuticals in the past year were the patent settlements with Teva Pharmaceuticals and Lupin Pharmaceuticals. These settlements resolve the major Paragraph IV challenges (a legal claim that a generic drug will not infringe on existing patents, or that the patents are invalid) that threatened immediate generic competition for Firdapse.

The core takeaway for investors is that both Teva Pharmaceuticals and Lupin Pharmaceuticals are now contractually barred from launching a generic version of Firdapse in the U.S. until February 25, 2035, at the earliest. This ten-year extension of market exclusivity is a massive de-risking event, protecting a substantial portion of the company's projected $545 Million to $565 Million in total revenue guidance for the full-year 2025. The settlements were achieved by granting a license that only becomes effective on that future date.

Pending patent litigation against Hetero USA Incorporated is set for trial in March 2026

Despite the successful settlements, the patent litigation against Hetero USA Incorporated remains a live risk. Hetero is challenging all of Firdapse's Orange Book-listed patents, which currently have expiration dates ranging from June 2032 to February 2037. This is the last major generic challenger standing.

Here's the quick math on the current litigation status:

• The lawsuit, filed in the U.S. District Court for the District of New Jersey, triggered an automatic 30-month stay on the FDA's ability to approve Hetero's Abbreviated New Drug Application (ANDA).
• This regulatory stay is currently set to expire in May 2026.
• The outcome of this litigation will determine if Catalyst Pharmaceuticals can secure the same 2035 exclusivity date against Hetero or if a generic could launch sooner.

Strengthened LEMS market control by acquiring rights to rival drug Ruzurgi in 2022

Catalyst Pharmaceuticals solidified its control over the LEMS treatment market by acquiring the U.S. and Mexico rights to the rival drug Ruzurgi from Jacobus Pharmaceutical Company, Inc. in July 2022. This move was a strategic masterstroke, converting a competitive threat into a proprietary asset.

The acquisition followed a favorable court ruling that found Ruzurgi's approval violated Catalyst's orphan drug exclusivity for Firdapse. The financial terms included a cash payment to Jacobus and a low single digit royalty on U.S. net sales of amifampridine. The immediate financial impact was noted as a one-time charge of $4.1 million related to the acquisition in the third quarter of 2022 GAAP Net Income, which is a small price to pay for eliminating the sole market competitor.

This consolidation effectively gives Catalyst Pharmaceuticals a near-monopoly on the LEMS treatment landscape in the U.S.

Ongoing risk of Paragraph IV challenges against Firdapse patents

While the settlements have bought significant time, the underlying legal mechanism-the Paragraph IV challenge-remains an ongoing risk for any blockbuster drug. The pharmaceutical industry is built on this 'patent dance.'

Firdapse is protected by six Orange Book-listed patents, but the very nature of a Paragraph IV challenge means that generic companies are actively looking for ways to argue that these patents are invalid or not infringed. The current litigation with Hetero is a perfect example of this persistent legal pressure. If the Hetero case goes poorly, the market could see a generic launch much earlier than the 2035 date secured through the other settlements.

The table below summarizes the current legal landscape for Firdapse's market protection:

Generic Challenger	Litigation Status (as of Nov 2025)	Generic Launch Date (Earliest)	Impact on Firdapse Exclusivity
Teva Pharmaceuticals	Settled	February 25, 2035	Exclusivity secured for a decade.
Lupin Pharmaceuticals	Settled	February 25, 2035	Exclusivity secured for a decade.
Hetero USA Incorporated	Ongoing Litigation	Earlier of court judgment or May 2026 (end of 30-month stay)	Primary near-term risk to market exclusivity.
Jacobus Pharmaceutical (Ruzurgi)	Acquired by Catalyst	Eliminated (Rights acquired in July 2022)	Market competition removed; LEMS market control solidified.

Next step: Your finance team should model the impact of a generic entry in late 2026 versus the expected 2035 date, using the $168.6 million first-half 2025 Firdapse revenue as the baseline for the at-risk revenue stream.

Catalyst Pharmaceuticals, Inc. (CPRX) - PESTLE Analysis: Environmental factors

Low operational environmental footprint due to outsourced manufacturing and a single office

Catalyst Pharmaceuticals operates with a naturally small direct environmental footprint, which is a key factor in its overall environmental profile. This low impact stems from its operating model: the company maintains only a single corporate office and relies entirely on outsourced manufacturing for its commercial products like FIRDAPSE (amifampridine) and AGAMREE (vamorolone). This approach shifts the most carbon-intensive activities-drug substance production, formulation, and packaging-to third-party contract manufacturing organizations (CMOs), reducing the company's direct Scope 1 and Scope 2 emissions (direct and purchased energy emissions) to a minimum.

Calculates and reports on Scope 1 and 2 greenhouse gas (GHG) emissions

Despite the limited direct footprint, Catalyst Pharmaceuticals is committed to transparency and actively calculates and reports its Scope 1 and Scope 2 greenhouse gas (GHG) emissions. This proactive step is crucial for establishing a baseline for future reduction targets. For the fiscal year 2024, the company's total reported emissions, including Scope 1, 2, and 3, amounted to 390.67 tCO2e (metric tons of carbon dioxide equivalent). The primary focus for direct reduction is the corporate headquarters, which has achieved an ENERGY STAR certification by using a state-of-the-art building automation system to optimize HVAC and lighting energy usage.

Negative impact noted in GHG Emissions from Amifampridine and clinical research services

While direct emissions are low, the environmental impact of the company's value chain, categorized as Scope 3 emissions (indirect emissions from the supply chain), is where the bulk of the footprint lies. The growth of key products like FIRDAPSE (amifampridine) and the expansion of clinical research services are the primary drivers here. The 2024 ESG Report noted that the overall Scope 3 GHG emissions increased compared to 2023. This increase was primarily attributed to the accelerated growth in Company operations and a rise in business travel, which is directly tied to commercial expansion and the oversight of clinical trials, such as the SUMMIT study for AGAMREE, which expanded to 19 clinical sites as of mid-2025.

Here's the quick math on the 2024 emissions breakdown, which illustrates the low direct impact:

GHG Scope Category	Description	2024 Emissions (tCO2e)
Scope 1	Direct emissions (e.g., company vehicles, owned facilities)	Included in 390.67 tCO2e total
Scope 2	Indirect emissions from purchased energy (electricity)	Included in 390.67 tCO2e total
Scope 3	Value chain emissions (e.g., outsourced manufacturing, business travel)	Majority of the 390.67 tCO2e total

Aligns with TCFD and SASB frameworks for environmental risk reporting

The company demonstrates a mature approach to climate risk by aligning its reporting with leading global frameworks. It has published its first disclosures aligned with the Task Force on Climate-related Financial Disclosures (TCFD) recommendations, which helps assess climate-related risks and opportunities. Also, the company's ESG program is informed by the Sustainability Accounting Standards Board (SASB) framework, ensuring that disclosures cover the material (most relevant) environmental topics for the biotechnology and pharmaceutical industry. This alignment signals to investors that the company is integrating climate strategy into its enterprise risk management (ERM).

Key actions driven by framework alignment:

• Conducted a climate-related risks and opportunities assessment.
• Committed to the Pledge to Net Zero, setting a 42% reduction target by 2030.
• Integrated a carbon tracking system, Obi, to enhance data accuracy.
• Purchases carbon offsets equivalent to its Scope 1 and business travel (Scope 3 Category 6) emissions.

Finance: Track the Q4 2025 earnings call for any changes to the $565 million-$585 million revenue guidance, specifically the impact of FYCOMPA generic competition.