Catalyst Pharmaceuticals Announces Sub-Licensee DyDo Pharma Launched FIRDAPSE® in Japan
Catalyst Pharmaceuticals (CPRX) announced that its sub-licensee DyDo Pharma has launched FIRDAPSE® (amifampridine) Tablets 10 mg in Japan. The drug is approved for improving muscle weakness in patients with Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder.
FIRDAPSE is the only FDA-approved therapy for LEMS treatment in adults and pediatric patients six years and older. This launch represents an expansion of Catalyst's geographic footprint and increases access to the therapy for Japanese patients. In the U.S., FIRDAPSE is supported by a comprehensive patient support program to ensure accessibility for eligible patients.
Catalyst Pharmaceuticals (CPRX) ha annunciato che il suo sub-concessionario DyDo Pharma ha lanciato le compresse FIRDAPSE® (amifampridina) da 10 mg in Giappone. Il farmaco è approvato per migliorare la debolezza muscolare nei pazienti affetti da sindrome miastenica di Lambert-Eaton (LEMS), un raro disturbo autoimmune.
FIRDAPSE è l'unica terapia approvata dalla FDA per il trattamento della LEMS in adulti e pazienti pediatrici di sei anni e oltre. Questo lancio rappresenta un'espansione della presenza geografica di Catalyst e aumenta l'accesso alla terapia per i pazienti giapponesi. Negli Stati Uniti, FIRDAPSE è supportata da un programma completo di assistenza ai pazienti per garantire l'accessibilità ai pazienti idonei.
Catalyst Pharmaceuticals (CPRX) anunció que su sublicenciatario DyDo Pharma ha lanzado FIRDAPSE® (amifampridina) en tabletas de 10 mg en Japón. El medicamento está aprobado para mejorar la debilidad muscular en pacientes con síndrome miasténico de Lambert-Eaton (LEMS), un raro trastorno autoinmune.
FIRDAPSE es la única terapia aprobada por la FDA para el tratamiento de LEMS en adultos y en pacientes pediátricos de seis años o más. Este lanzamiento representa una expansión de la presencia geográfica de Catalyst y aumenta el acceso a la terapia para los pacientes japoneses. En EE. UU., FIRDAPSE cuenta con un programa completo de apoyo a pacientes para garantizar el acceso a los pacientes elegibles.
Catalyst Pharmaceuticals (CPRX)는 그들의 서브 라이센시인 DyDo Pharma가 일본에서 FIRDAPSE® (아미팜프리딘) 10mg 정제를 출시했다고 발표했습니다. 이 약물은 램버트-이터 미안증후군 (LEMS) 환자의 근육 약화를 개선하도록 승인되었습니다. 이는 드문 자가 면역 질환입니다.
FIRDAPSE는 성인 및 6세 이상의 소아 환자에 대한 LEMS 치료를 위해 FDA의 승인을 받은 유일한 치료법입니다. 이번 출시는 Catalyst의 지리적 범위를 확장하고 일본 환자들의 치료 접근성을 높입니다. 미국에서는 FIRDAPSE가 자격이 있는 환자들이 접근할 수 있도록 포괄적인 환자 지원 프로그램을 지원합니다.
Catalyst Pharmaceuticals (CPRX) a annoncé que son sous-licencié DyDo Pharma a lancé les comprimés FIRDAPSE® (amifampridine) de 10 mg au Japon. Le médicament est approuvé pour améliorer la faiblesse musculaire chez les patients atteints de sydrome myasthénique de Lambert-Eaton (LEMS), un rare trouble auto-immun.
FIRDAPSE est la seule thérapie approuvée par la FDA pour le traitement de la LEMS chez les adultes et les patients pédiatriques de six ans et plus. Ce lancement représente une expansion de l'empreinte géographique de Catalyst et augmente l'accès à la thérapie pour les patients japonais. Aux États-Unis, FIRDAPSE est soutenue par un programme complet de soutien aux patients pour garantir l'accessibilité aux patients éligibles.
Catalyst Pharmaceuticals (CPRX) gab bekannt, dass ihr Sub-Lizenznehmer DyDo Pharma die FIRDAPSE® (Amifampridin) Tabletten zu 10 mg in Japan eingeführt hat. Das Medikament ist genehmigt, um die Muskelschwäche bei Patienten mit Lambert-Eaton Myasthenic Syndrome (LEMS), einer seltenen autoimmunen Erkrankung, zu verbessern.
FIRDAPSE ist die einzige von der FDA genehmigte Therapie zur Behandlung von LEMS bei Erwachsenen und pädiatrischen Patienten ab sechs Jahren. Diese Einführung stellt eine Erweiterung des geografischen Fußabdrucks von Catalyst dar und erhöht den Zugang zur Therapie für japanische Patienten. In den USA wird FIRDAPSE durch ein umfassendes Patientenunterstützungsprogramm unterstützt, um die Zugänglichkeit für berechtigte Patienten zu gewährleisten.
- Geographic expansion into Japanese market through sub-licensee DyDo Pharma
- Maintains position as only FDA-approved therapy for LEMS treatment
- None.
Insights
The launch of FIRDAPSE in Japan through DyDo Pharma represents a strategic market expansion for Catalyst Pharmaceuticals that opens up the world's third-largest pharmaceutical market. Japan's rare disease market is particularly attractive due to its aging population and favorable pricing policies for orphan drugs. The Japanese pharmaceutical market is valued at approximately
This launch should generate a steady revenue stream through royalties from DyDo, though initial contribution may be modest given LEMS' rare disease status with an estimated prevalence of 3 per million in Japan. Based on these demographics, the addressable patient population in Japan would be approximately 350-400 patients. Given typical Japanese orphan drug pricing, this could translate to an annual market opportunity of
The successful regulatory pathway in Japan also validates FIRDAPSE's clinical profile and could facilitate additional geographic expansions. Having secured commercialization in key markets - US, Europe and now Japan - Catalyst has established a strong global presence for its lead asset.
The introduction of FIRDAPSE to the Japanese market is particularly significant from a clinical perspective. LEMS patients in Japan previously had treatment options, primarily relying on off-label medications or symptomatic treatments. FIRDAPSE's mechanism of action as a voltage-gated potassium channel blocker provides targeted therapy that directly addresses the underlying neuromuscular transmission defect in LEMS.
The Japanese LEMS patient community will benefit from access to a treatment that has demonstrated consistent efficacy in clinical trials, showing improvements in Quantitative Myasthenia Gravis (QMG) scores and Subject Global Impression (SGI) ratings. The drug's safety profile is well-established through years of post-marketing surveillance in other regions, which should provide confidence to Japanese healthcare providers.
In simpler terms: Think of LEMS as a faulty electrical connection between nerves and muscles. FIRDAPSE acts like a signal booster, helping strengthen these connections so muscles can work properly again. This launch means Japanese patients now have access to a proven "signal booster" rather than relying on less targeted treatments.
CORAL GABLES, Fla., Jan. 21, 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 diseases, today reported that its sub-licensee in Japan, DyDo Pharma, Inc., ("DyDo") has launched FIRDAPSE® (amifampridine) Tablets 10 mg in Japan for the indication of improving muscle weakness in patients living with Lambert-Eaton myasthenic syndrome ("LEMS").
"We are pleased that our sub-licensee, DyDo, has launched FIRDAPSE in Japan. We believe in health equity, and this launch is a testament to our ongoing efforts to increase patient access to life-changing therapies worldwide,” said Richard J. Daly, Catalyst’s President and Chief Executive Officer. “The launch of FIRDAPSE in Japan marks another advancement in our efforts to expand the geographic footprint of our portfolio products and paves the way for healthcare providers and patients in Japan to access this therapy."
FIRDAPSE (amifampridine) is the only U.S. FDA approved, evidence-based therapy for the treatment of LEMS in adults and pediatric patients six years of age and older. LEMS is a rare autoimmune disorder characterized by muscle weakness and fatigue. As a cornerstone of Catalyst's commitment to serving those with rare diseases, FIRDAPSE is supported in the U.S. by a comprehensive patient support program to help ensure accessibility and assistance for eligible U.S. patients.
About FIRDAPSE® (amifampridine) Tablets 10 mg
FIRDAPSE® (amifampridine) Tablets 10 mg is an oral, nonspecific, voltage-dependent, potassium (K+) channel blocker that causes depolarization of the presynaptic membrane and slows or inhibits repolarization. This action results in the opening of slow voltage-dependent calcium (Ca2+) channels, allowing for a subsequent influx of Ca2+. In turn, it induces the exocytosis of synaptic vesicles containing acetylcholine (ACh) to release more ACh into the synaptic cleft, enhancing neuromuscular transmission and providing for improved muscle function. Amifampridine phosphate was granted orphan drug designation by the Ministry of Health, Labor, and Welfare in Japan, and FIRDAPSE has previously been approved for use in the U.S. in adults and pediatric patients six years of age and older and in Europe and Canada for the treatment of adults with LEMS.
For Full Prescribing and Safety Information for FIRDAPSE, please visit www.firdapse.com.
About DyDo Pharma
DyDo Pharma is the rare disease pharmaceutical wholly-owned subsidiary of DyDo Group Holdings. DyDo Group Holdings, Inc. operates through the following segments: Domestic Beverage Business, International Beverage Business, Pharmaceutical-related Business, Food Business, and Pharmaceutical Business. The Domestic Beverage Business accounts for more than
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 while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, FL., was recognized as one of North America's Fastest-Growing Companies on the 2024 Deloitte Technology Fast 500™ List.
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 (i) whether DyDo can successfully commercialize FIRDAPSE in Japan, (ii) whether Catalyst's revenues derived in future periods from its sub-license with DyDo will be material to Catalyst, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2023, its Quarterly Report on Form 10-Q for the fiscal quarter ended September 30, 2024, and its other 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.
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