Catalyst Pharmaceuticals

Name

Catalyst Pharmaceuticals

Legal name

Catalyst Pharmaceuticals, Inc.

Website

https://catalystpharma.com

Company type

Public

Founded year

2002

Operating status

Operating

Headcount range

51–100 employees

Ownership category

akta.pro rank

Product category

Specialty Pharmaceuticals - Rare Disease Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Pharmaceutical and Medicine Manufacturing (3254)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• Rare disease therapeutics
• Neuromuscular disease treatments
• Specialty pharmaceuticals
• Orphan drug commercialization
• Biopharmaceutical therapies

Headquarters

HQ city

Coral Gables

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Marketing or Sales, Operations, Technology or R&D, Supply Chain

Revenue model

1. FIRDAPSE (amifampridine) Sales: Revenue from sales of FIRDAPSE Tablets 10 mg for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). FIRDAPSE is the only FDA-approved evidence-based treatment for LEMS, generating $358.4 million in 2025 revenue with 17.1% year-over-year growth.
2. AGAMREE (vamorolone) Sales: Revenue from sales of AGAMREE oral suspension 40 mg/mL for Duchenne muscular dystrophy. Launched in the US in March 2024, generating $117.1 million in 2025 with 154.3% year-over-year growth. Royalty rates declined from up to 18.5% to 6% effective January 26, 2026 following expiration of certain tiered royalties.
3. FYCOMPA (perampanel) Sales: Revenue from sales of FYCOMPA for partial-onset seizures and primary generalized tonic-clonic seizures. Acquired US rights from Eisai in January 2023. Revenue declined 61.3% following loss of exclusivity in 2025 due to generic competition.

Pricing tiers

Go-to-market motion2 records

Distribution channels5 records

Marketing channels7 records

Core offering

Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company that in-licenses, develops, and commercializes novel prescription medicines for rare and difficult-to-treat diseases. Its portfolio consists of three FDA-approved branded products: FIRDAPSE (amifampridine) for Lambert-Eaton Myasthenic Syndrome, AGAMREE (vamorolone) for Duchenne Muscular Dystrophy, and FYCOMPA (perampanel) for epilepsy, distributed through specialty pharmacy channels.

Product overview

Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for rare and difficult-to-treat diseases. The company operates a portfolio of three FDA-approved products: FIRDAPSE (amifampridine) for Lambert-Eaton Myasthenic Syndrome (LEMS), AGAMREE (vamorolone) for Duchenne Muscular Dystrophy (DMD), and FYCOMPA (perampanel) for epilepsy. The company generates revenue through commercializing these branded pharmaceuticals in the United States, with additional expansion into Canada and Japan. Catalyst's strategy centers on rare neuromuscular and neurological diseases, with products addressing significant unmet medical needs in specialized patient populations. In 2025, the company reported $589 million in total revenues, with FIRDAPSE contributing $358.4 million and AGAMREE contributing $117.1 million, while FYCOMPA faced generic competition following loss of exclusivity. The company has agreed to be acquired by Angelini Pharma for approximately $4.1 billion.

Differentiator

Problem solved

Functional benefit

Brands

• AGAMREE: Vamorolone oral suspension 40 mg/mL - treatment for Duchenne muscular dystrophy (DMD) in patients 2 years of age and older
• FIRDAPSE
• FYCOMPA
• Catalyst Pathways
• Catalyst Convos

Products and services

• FIRDAPSE (amifampridine) Tablets 10 mg FIRDAPSE is the only FDA-approved treatment for Lambert-Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disease affecting neuromuscular junction function. Available for patients 6 years of age and older, it works by stimulating acetylcholine release to improve muscle function and mobility.
• AGAMREE (vamorolone) oral suspension 40 mg/mL AGAMREE is a novel corticosteroid with a unique structure that retains anti-inflammatory effects for Duchenne Muscular Dystrophy (DMD) while showing less pronounced immunosuppressive effects compared to traditional corticosteroids. FDA approved for patients 2 years of age and older.
• FYCOMPA (perampanel) FYCOMPA is an antiseizure medication used alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures in people aged 4 and older, and with other medicines to treat primary generalized tonic-clonic seizures in people aged 12 and older.
• Catalyst Pathways Patient Support Program Catalyst Pathways is a premier patient support program offering everything from disease education to financial support, helping eligible patients access Catalyst therapies regardless of ability to pay.

Quantifiable outcome

• AGAMREE demonstrated 80% reduction in vertebral fractures compared to deflazacort (8.1% vs 41.9%) over 8 years of treatment
• +5 more outcomes

Named customers7 records

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Ten partnerships are on record, tiered core, minor and major.

• Angelini Pharma S.p.A.coreStrategic or Co-development Partner · 7 May 2026Angelini Pharma agreed to acquire Catalyst Pharmaceuticals for approximately $4.1 billion USD (€3.5 billion), or $31.50 per share in cash, representing a 28% premium to the 30-day volume-weighted average trading price. The acquisition marks Angelini Pharma's entry into the U.S. market and is expected to close in Q3 2026, subject to Catalyst stockholder approval and regulatory clearances. Upon closing, Angelini Pharma intends to integrate Catalyst's rare disease portfolio with its Brain Health expertise.
• Florida Society of Clinical Oncology (FLASCO)minorStrategic or Co-development Partner · 7 April 2026Corporate partnership with FLASCO aimed at providing education, timely dissemination of pertinent information, and general support to Florida oncology healthcare professionals.
• BioFloridaminorStrategic or Co-development Partner · 7 April 2026Catalyst Pharmaceuticals is an important part of Florida's life sciences sector for almost 25 years. Active in BioFlorida events and ecosystem.
• Miami Biotech CollectiveminorStrategic or Co-development Partner · 7 April 2026Catalyst participates in Miami Biotech Collective ecosystem connecting industry, academia, and healthcare systems.
• University of Notre DameminorStrategic or Co-development Partner · 1 January 2026Inaugural Catalyst Advocacy Scholars Summit (CASS) hosting 12 scholars from University of Notre Dame for immersive learning experience in rare disease patient advocacy. Students participated in presentations from various departments and capstone projects on DMD and LEMS.
• Cure DuchenneminorStrategic or Co-development Partner · 1 January 2026Patient advocacy organization partnership. CEO & Founder Debra Miller presented at CASS program about the organization's journey.
• KIF1A.org, Inc.minorStrategic or Co-development Partner · 1 January 2026Patient advocacy organization co-founded by Luke Rosen. Luke Rosen participated in CASS program. Organization focused on KIF1A neurological disorders.
• Lipodystrophy UnitedminorStrategic or Co-development Partner · 1 January 2026Patient advocacy organization. Chief Advisor, Board Member, and Co-Founder Andra Statton participated in CASS program.
• DyDo Pharma, Inc.majorChannel Partner/ Reseller/ Distributor · 1 June 2021Exclusive license and supply agreement for development and commercialization of FIRDAPSE Tablets 10 mg in Japan for treatment of LEMS. Japan MHLW approved FIRDAPSE in September 2024; FIRDAPSE became commercially available in Japan in January 2025.
• KYE PharmaceuticalsmajorChannel Partner/ Reseller/ Distributor · 1 August 2020Sublicensed rights to KYE Pharmaceuticals for commercialization of FIRDAPSE in Canada (approved 2020) and AGAMREE (Health Canada approved October 2, 2025 for boys age 4 and older). Catalyst supplies product to KYE who handles regulatory approval and commercialization in Canada.

Scale indicators12 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Sarepta Therapeutics: Direct DMD competitor marketing Elevidys (delandistrogene moxeparvovec) gene therapy. Both companies compete in the Duchenne muscular dystrophy space targeting similar patient populations (boys 2+ years old), with vamorolone and Elevidys positioned as alternative treatment options.
• PTC Therapeutics: Direct DMD competitor with Translarna (ataluren) and Emflaza (deflazacort). Emflaza is the incumbent corticosteroid that AGAMREE/vamorolone directly challenges, making PTC one of Catalyst's most relevant competitive benchmarks in the rare neuromuscular space.
• Santhera Pharmaceuticals: Originator and licensor of AGAMREE/vamorolone, holding ex-North America rights. Comparable rare-disease specialty pharma with similar DMD focus, royalty-driven revenue model, and overlap in neuromuscular indications targeted by Catalyst's pipeline.
• Ultragenyx Pharmaceutical: Commercial-stage rare-disease biopharma with comparable small-patient-population model, in-licensing strategy, and specialty distribution. Comparable in scale, headcount, and focus on rare neuromuscular and metabolic diseases with FDA-approved therapies.
• BioMarin Pharmaceutical: Original licensor of FIRDAPSE to Catalyst and a major rare-disease player with Vimizim, Naglazyme, and Voxzogo. Highly comparable as an established rare-disease commercializer with similar regulatory expertise and patient-access infrastructure.

Broad incumbents

• Jazz Pharmaceuticals: Larger specialty biopharma with rare neurology and oncology franchises (Xywav, Epidiolex). Comparable commercial model and rare neurology overlap with FYCOMPA, while broader portfolio and significantly larger scale make it a broad incumbent benchmark.
• Alexion (AstraZeneca Rare Disease): Industry-leading rare-disease unit with Soliris/Ultomiris, Strensiq, and Koselugo. Directly comparable Catalyst Pathways-style patient support model and rare neuromuscular/neurology focus, but with much broader portfolio and global reach.
• Recordati Rare Diseases: European-based rare-disease specialty pharma with comparable in-licensing and commercialization model. Overlaps in rare neurological and metabolic conditions and shares the Angelini/Italian-pharma ecosystem context.
• Lundbeck: Established neurology-focused pharma with rare epilepsy and movement disorder treatments. Highly comparable commercial focus on neurology specialists and similar rare neurology go-to-market motion, but at meaningfully larger scale.

Others

• Eisai Co., Ltd. Originated and divested FYCOMPA rights to Catalyst. Not a direct competitor but a critical counterparty/ecosystem player; comparable in CNS pipeline focus and rare neurology development capability.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/catalystpharma
• Facebookfacebook.com/CatalystForRare
• LinkedInlinkedin.com/company/catalystforrare
• Instagraminstagram.com/catalystforrare

Revenue estimate

Valuation estimate

Number of profiles

Profiles17 records

Funding overview

Funding rounds7 records

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Catalyst Pharmaceuticals