Lambert Eaton Myasthenic Syndrome (LEMS) Antibody Testing and Treatment Recommendations Added to NCCN Clinical Practice Guidelines for Small Cell Lung Cancer (SCLC)
Catalyst Pharmaceuticals (NASDAQ:CPRX) announced that the National Comprehensive Cancer Network (NCCN) has updated its Clinical Practice Guidelines for Small Cell Lung Cancer (SCLC) to include important recommendations for Lambert Eaton Myasthenic Syndrome (LEMS).
The updated guidelines now include testing for PQ- and N-type voltage-gated calcium channel (VGCC) antibodies and recommend neurological evaluation for diagnosis. Additionally, amifampridine (FIRDAPSE®) is now recommended as a potential treatment option in consultation with neurology.
Notably, approximately 50% of LEMS cases are associated with underlying malignancy, primarily SCLC, with LEMS observed in 3% of SCLC patients. Internal analysis suggests that 90% of LEMS patients with SCLC may be undiagnosed, highlighting a significant unmet medical need.
Catalyst Pharmaceuticals (NASDAQ:CPRX) ha annunciato che la National Comprehensive Cancer Network (NCCN) ha aggiornato le sue Linee Guida Cliniche per il Carcinoma Polmonare a Piccole Cellule (SCLC), includendo raccomandazioni importanti per la Sindrome di Lambert-Eaton Miastenica (LEMS).
Le linee guida aggiornate prevedono ora il test per gli anticorpi contro i canali del calcio voltaggio-dipendenti di tipo PQ e N (VGCC) e raccomandano una valutazione neurologica per la diagnosi. Inoltre, l'amifampridina (FIRDAPSE®) è ora suggerita come possibile opzione terapeutica in consultazione con uno specialista in neurologia.
È importante sottolineare che circa il 50% dei casi di LEMS è associato a una neoplasia sottostante, principalmente SCLC, con LEMS osservata nel 3% dei pazienti con SCLC. Un’analisi interna indica che il 90% dei pazienti con LEMS e SCLC potrebbe non essere diagnosticato, evidenziando un significativo bisogno medico insoddisfatto.
Catalyst Pharmaceuticals (NASDAQ:CPRX) anunció que la National Comprehensive Cancer Network (NCCN) ha actualizado sus Guías de Práctica Clínica para el Cáncer de Pulmón de Células Pequeñas (SCLC), incluyendo recomendaciones importantes para el Síndrome Miasténico de Lambert-Eaton (LEMS).
Las guías actualizadas ahora incluyen la prueba de anticuerpos contra los canales de calcio dependientes de voltaje tipo PQ y N (VGCC) y recomiendan una evaluación neurológica para el diagnóstico. Además, se recomienda amifampridina (FIRDAPSE®) como una posible opción de tratamiento en consulta con neurología.
Es relevante destacar que aproximadamente el 50% de los casos de LEMS están asociados con una malignidad subyacente, principalmente SCLC, con LEMS observado en el 3% de los pacientes con SCLC. Un análisis interno sugiere que el 90% de los pacientes con LEMS y SCLC podrían no estar diagnosticados, lo que resalta una necesidad médica importante no satisfecha.
Catalyst Pharmaceuticals (NASDAQ:CPRX)는 National Comprehensive Cancer Network (NCCN)가 소세포폐암(SCLC)에 대한 임상 진료 지침을 업데이트하여 램버트 이튼 근무력증 증후군(LEMS)에 대한 중요한 권고사항을 포함했다고 발표했습니다.
업데이트된 지침에는 이제 PQ형 및 N형 전압 의존성 칼슘 채널(VGCC) 항체 검사가 포함되었으며 진단을 위한 신경학적 평가를 권장합니다. 또한, 아미팜프리딘(FIRDAPSE®)이 신경과 상담 하에 잠재적 치료 옵션으로 권고됩니다.
특히, 약 50%의 LEMS 사례가 기저 악성 종양과 연관되어 있으며 주로 SCLC에서 발생하며, SCLC 환자의 3%에서 LEMS가 관찰됩니다. 내부 분석에 따르면 SCLC 환자 중 90%의 LEMS 환자가 진단되지 않았을 가능성이 있어 상당한 미충족 의료 수요를 강조합니다.
Catalyst Pharmaceuticals (NASDAQ:CPRX) a annoncé que le National Comprehensive Cancer Network (NCCN) a mis à jour ses lignes directrices cliniques pour le cancer du poumon à petites cellules (SCLC) afin d'inclure des recommandations importantes pour le Syndrome de Lambert-Eaton (LEMS).
Les lignes directrices mises à jour incluent désormais le test des anticorps contre les canaux calciques voltage-dépendants de type PQ et N (VGCC) et recommandent une évaluation neurologique pour le diagnostic. De plus, l’amifampridine (FIRDAPSE®) est désormais recommandée comme option thérapeutique potentielle en consultation avec un neurologue.
Il est à noter qu'environ 50 % des cas de LEMS sont associés à une malignité sous-jacente, principalement le SCLC, avec une incidence de LEMS chez 3 % des patients atteints de SCLC. Une analyse interne suggère que 90 % des patients atteints de LEMS et de SCLC pourraient ne pas être diagnostiqués, soulignant un besoin médical important non satisfait.
Catalyst Pharmaceuticals (NASDAQ:CPRX) gab bekannt, dass das National Comprehensive Cancer Network (NCCN) seine klinischen Leitlinien für kleinzelligen Lungenkrebs (SCLC) aktualisiert hat, um wichtige Empfehlungen für das Lambert-Eaton-Myasthenie-Syndrom (LEMS) aufzunehmen.
Die aktualisierten Leitlinien beinhalten nun Tests auf PQ- und N-Typ spannungsabhängige Calciumkanal-Antikörper (VGCC) und empfehlen eine neurologische Untersuchung zur Diagnose. Zudem wird Amifampridin (FIRDAPSE®) als mögliche Behandlungsoption in Absprache mit der Neurologie empfohlen.
Bemerkenswert ist, dass etwa 50 % der LEMS-Fälle mit einer zugrunde liegenden Krebserkrankung, hauptsächlich SCLC, verbunden sind, wobei LEMS bei 3 % der SCLC-Patienten beobachtet wird. Interne Analysen deuten darauf hin, dass 90 % der LEMS-Patienten mit SCLC möglicherweise nicht diagnostiziert werden, was einen erheblichen ungedeckten medizinischen Bedarf aufzeigt.
- None.
- High rate of undiagnosed LEMS cases (90%) indicates significant current underdiagnosis
- Complex diagnostic process requiring both neurological consultation and specific antibody testing
Insights
NCCN guideline inclusion of LEMS testing and amifampridine significantly validates Catalyst's treatment for an underdiagnosed condition in cancer patients.
This guideline update represents a significant clinical validation for Catalyst Pharmaceuticals' FIRDAPSE (amifampridine). The National Comprehensive Cancer Network (NCCN) Guidelines are the gold standard for clinical decision-making in oncology, used by approximately 90% of U.S. cancer centers. Their inclusion of specific LEMS testing and treatment recommendations creates a clear diagnostic and treatment pathway that previously didn't exist for many patients.
The statistics are particularly compelling: with 3% of SCLC patients developing LEMS and an estimated 90% going undiagnosed, there's a substantial population of patients suffering unnecessarily. These patients experience debilitating proximal muscle weakness while already battling cancer, potentially compromising their quality of life and possibly even treatment outcomes.
From a clinical adoption perspective, the specific recommendation for VGCC antibody testing with neurological consultation creates a definitive diagnostic protocol. More importantly, the guidelines explicitly name amifampridine as a treatment to be considered, rather than just suggesting symptom management or a general drug class.
This rare disease recognition within cancer care represents a paradigm shift in how supportive care is approached for SCLC patients. By addressing comorbid neurological conditions like LEMS, the updated guidelines acknowledge that comprehensive cancer treatment must consider quality of life factors alongside primary disease management. For LEMS patients battling cancer, this increased awareness could mean the difference between debilitating weakness and maintained functionality during cancer treatment.
- VGCC antibody testing in conjunction with neurologic consultation can diagnose LEMS
- Amifampridine may be considered as a supportive care treatment
- Guideline inclusion may raise clinical awareness of cancer-associated LEMS to support broader VGCC diagnostic testing and treatment adoption
CORAL GABLES, Fla., Aug. 06, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today announced the National Comprehensive Cancer Network® (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Small Cell Lung Cancer (SCLC) now include new additions involving Lambert Eaton myasthenic syndrome (LEMS), amifampridine (FIRDAPSE®), and the tests for PQ- and N-type voltage-gated calcium channel (VGCC) antibodies.
The updated NCCN Clinical Practice Guidelines in Oncology for SCLC relating to LEMS now include symptom specificity—characterized by proximal muscle weakness and autonomic dysfunction. Under “Signs and Symptoms of Small Cell Lung Cancer” (SCL-A 2 of 2), the guidelines recommend diagnosis through a neurological evaluation, ideally in consultation with a neurologist, which may include testing for PQ- and N-type VGCC antibodies. Additionally, under “Principles of Supportive Care” (SCL-D), the guidelines recommend that amifampridine should be considered as a treatment in consultation with neurology.1
Approximately 50 percent of LEMS cases are associated with an underlying malignancy, most commonly SCLC. Literature suggests that LEMS is observed in 3 percent of SCLC patients. Based on an internal healthcare database analysis of SCLC claims, potentially 90 percent of LEMS patients with SCLC went undiagnosed. These undiagnosed LEMS patients may suffer from LEMS symptoms while undergoing treatment for SCLC.
“Early diagnosis of LEMS in SCLC is critical, as it may enable patients to have better outcomes if their LEMS symptoms are effectively treated while fighting SCLC. Accurate identification through VGCC antibody testing and comprehensive neurological evaluation is essential,” said William Andrews, MD, FACP, Chief Medical Officer, Catalyst. “The NCCN Guidelines are a trusted standard for guiding treatment decisions, and we believe this update will drive greater awareness. Ultimately, it will help patients, caregivers, and healthcare providers make more informed choices when addressing this serious unmet need.”
1NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. To learn more about NCCN go to https://www.nccn.org/home/about
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX), is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence, which remains the cornerstone of our commercial strategy, while continuously evaluating strategic opportunities to expand our global footprint. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies.
For more information, please visit Catalyst's website at www.catalystpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2024 and its subsequent filings with the U.S. Securities and Exchange Commission (“SEC”), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
Source: Catalyst Pharmaceuticals, Inc.
Investor Contact Mary Coleman, Catalyst Pharmaceuticals, Inc. (305) 420-3200 ir@catalystpharma.com Media Contact David Schull, Russo Partners (858) 717-2310 david.schull@russopartnersllc.com
FAQ
What changes did NCCN make to its SCLC guidelines regarding LEMS in August 2025?
How common is LEMS in Small Cell Lung Cancer (SCLC) patients?
What is the current diagnosis rate for LEMS in SCLC patients according to Catalyst Pharmaceuticals?
What treatment does NCCN recommend for LEMS in SCLC patients?
How is LEMS diagnosed according to the new NCCN guidelines?
