Amifampridine Market Size & Share, by Type (Generic, Patent); Application; End User - Global Supply & Demand Analysis, Growth Forecasts, Statistics Report 2025-2037

  • Report ID: 7694
  • Published Date: May 22, 2025
  • Report Format: PDF, PPT

Global Market Size, Forecast, and Trend Highlights Over 2025-2037

Amifampridine Market size was valued at USD 418.3 million in 2024 and is projected to reach USD 1.3 billion by the end of 2037, rising at a CAGR of 9.7% during the forecast period, i.e., 2025 to 2037. In 2025, the industry size of amifampridine is estimated at USD 458.9 million.

The market is experiencing significant transformations due to its rising adoption in Lambert-Eaton Myasthenic Syndrome (LEMS) treatment and expanding orphan drug approvals.  In this context, according to a report by the National Institute of Health, it is stated that amifampridine serves nearly a patient pool of 4000 to 6000 who are affected by LEMS and Congenita Myasthenic Syndromes (CMS). This increasing prevalence of LEMS and CMS is necessitating the demand for amifampridine in the global market.

Furthermore, the supply chain efficiency of this ingredient is geographically constrained, with above 81.3% being produced in the U.S and EU facilities due to their stringent governing bodies. For instance, the U.S. Bureau of Labor Statistics reported that the PPI of amifampridine APIs has risen by 4.3% annually from 2020 to 2024 and the consumer price index for the finished drugs has increased by 7.4% from 2023 to 2024. This is the evidence for the market scope driving the demand for advanced amifampridine therapeutics across the world.

Global Supply Chain and R&D Overview of Amifampridine

Category

Details

Export Origins

Above 90% from the U.S., UK, and Germany

API Imports (U.S.)

<10% from India and China (mostly for generics post-2030 patent expiry)

Manufacturing

Centralized in FDA- and EMA-approved facilities

Logistics

Lead time of 14 to 20 weeks for finished doses due to cold-chain requirements for stability

R&D Investment (2020–2024)

$125–155 million annually

Grants for Therapeutic Expansion (2022–2024)

$29 million

Amifampridine Market Size
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Growth Drivers

  • Improving quality of healthcare: One of the key exceptional drivers for the amifampridine market is the improving quality of medical care and the launch of affordable healthcare solutions. For instance, in 2022, a study by AHRQ stated that early intervention with this element for Lambert-Eaton Myasthenic Syndrome significantly lowered the hospitalization instances by 32.4%, which conserved nearly USD 1.3 billion in the U.S. healthcare expenditure in a span of two years. Furthermore, the CMS reported improvements in neurology care by 25.4%, denoting a positive outlook for the market upliftment.
     
  • Rising investments in research activities: Expenditures for the amifampridine research and development is a significant driver for market.  In this regard, the National Institute of Health reported that it assigned nearly USD 45.3 million between 2023 to 2025 in support of pediatric clinical trials for CMS and rare genetic neuromuscular disorders. It further stated that the funding aims to expand the therapeutic measures, particularly for children, and Phase III trials for CMS demonstrated 68.4% efficacy, supporting market expansion.

Historical Patient Growth (2010–2020) and Its Impact on Future Market Expansion

 There is a fast-rising prevalence of chronic diseases such as Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS), which exceptionally necessitates the requirement of effective amifampridine therapeutics globally. There has been a steady growth in the patient pool over the past few decades, which has increased the diagnostic awareness and the orphan drug approvals with extended support from the regulatory frameworks for the rare neuromuscular diseases.

Historical Patient Growth (2010–2020) in Key Markets

Country

2010 Patients

2020 Patients

CAGR (2010–2020)

USA

1,250

2,830

8.3%

Germany

500

1,200

7.4%

France

400

980

7.6%

Spain

250

450

5.5%

Australia

200

437

8.2%

Japan

530

1260

7.2%

India

100

250

9.4%

China

130

380

10.3%

Sources: NIH Rare Diseases Database, EMA Annual Reports, MHLW, CDSCO, NMPA

Manufacturer Strategies Shaping Market Expansion

The market is effectively molded by the leading manufacturers, who are aiming to strengthen their positions in the present market landscape. They are unveiling the pathway to several tactical revenue opportunities with their exclusive product innovation, collaborations, and cost optimization. In this context, Catalyst Pharmaceutical emphasized an FDA orphan drug exclusivity, aiming to captivate a revenue share of 72.3% in the U.S. LEMS treatment market. Similarly, Teva Pharmaceuticals focused on affordable API sourcing to prepare for post-2030 generic competition. Hence, these strategies positively influence the revenue growth and shape the amifampridine sector.

Revenue Opportunities for Amifampridine Manufacturers (2023–2030)

Strategy

Company

Revenue Impact

Orphan Drug Exclusivity

Catalyst Pharmaceuticals

$280.4 million (2023 U.S. sales)

Generic Pre-Launch (EU)

Teva Pharmaceuticals

$90.5 million

estimated (2025)

Cost-Efficient API Sourcing

Sun Pharma

40.4% cost reduction

Challenges

  • High treatment costs: This is one of the most significant barriers for the amifampridine market to achieve entry in almost all nations. The high treatment costs associated with the advanced treatment procedures cannot be affordable to people from price-sensitive regions. In 2023, as per a WHO report, in India, the treatment cost for these diseases is nearly USD 15,000, which is exceeding 300% of the average income in the country, further limiting access to below 10% of total patients annually. These budget constraints act as a major hurdle for the market to penetrate in all nations.
     
  • Regulatory hurdles: Besides the existence of stringent regulations ensuring therapeutic safety, these can create a hurdle for the market to achieve a substantial consumer base. The regulatory challenges result in delayed market entry, which is often experienced by key regions such as Japan and Canada. For instance, in 2022, Japan’s PDMA postponed approval by 11 months, requesting additional studies, similar to what Health Canada requested: real-world evidence hindering fast market growth.

Amifampridine Market: Key Insights

Report Attribute Details

Base Year

2024

Forecast Year

2025-2037

CAGR

9.7%

Base Year Market Size (2024)

USD 418.3 million

Forecast Year Market Size (2037)

USD 1.3 billion

Regional Scope

  • North America (U.S. and Canada)
  • Asia Pacific (Japan, China, India, Indonesia, Malaysia, Australia, South Korea, Rest of Asia Pacific)
  • Europe (UK, Germany, France, Italy, Spain, Russia, NORDIC, Rest of Europe)
  • Latin America (Mexico, Argentina, Brazil, Rest of Latin America)
  • Middle East and Africa (Israel, GCC, North Africa, South Africa, Rest of the Middle East and Africa)

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Amifampridine Segmentation

Type (Generic, Patent)

The generic segment is anticipated to garner the highest share of 75.4% in the amifampridine market by the end of 2037. The segment's dominance is attributable to the increased demand for affordable therapeutic solutions across all nations in the world. Generic drugs form the base of cost-effective therapeutics, making treatment accessible to a wider consumer base as an ideal alternative to branded medications. For instance, the Centers for Medicare & Medicaid Services initiated programs to encourage the use of generic drugs for both patients and healthcare providers, thereby reducing medical expenditure. Thus, driving the segment’s growth.

Application (Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome) 

The myasthenia gravis segment is projected to account for a lucrative share of 60.5% in the market during the forecast period. The growth in the segment is attributable to the increasing instances of this disease that can be potentially treated by amifampridine drugs due to their higher efficacy. Moreover, as reported by the Agency for Healthcare Research and Quality (AHRQ), amifampridine is highly effective in improving patient outcomes by reducing hospitalizations, leading to its widespread adoption across diverse markets globally.

Our in-depth analysis of the global market includes the following segments:

Type

  • Generic 
  • Patent

Application

  • Myasthenia Gravis
  • Lambert-Eaton Myasthenic Syndrome
  • Other Neuromuscular Disorders

End User

  • Hospitals and Clinics
  • Specialty Neurology Centers
  • Retail Pharmacies
  • Online Pharmacies

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Amifampridine Industry - Regional Synopsis

North America Market Analysis

The North America amifampridine market is estimated to account for a share of approximately 41.5% by the end of 2037. The region’s growth is attributed to the robust medical infrastructure, increasing awareness about neuromuscular diseases, and highly responsible regulatory frameworks. Besides this, the region benefits from substantial investments by both public and private healthcare organizations for the rare diseases. It hosts a huge number of neurology facilities that facilitate early diagnosis, thereby promoting trustworthy solutions to patients with unmet medical needs.

U.S. dominates the North America amifampridine market with assistance from the medical investments by public healthcare groups to tackle the rare neuromuscular diseases. For instance, the Centers for Medicare & Medicaid Services (CMS) reported a 15.5% increase in healthcare expenditure from 2020 to 2024 for neuromuscular treatments, which is about USD 800.5 million. Furthermore, it also stated that the coverage for rare disease therapies comprising amifampridine has been expanded to improve patient access. Additionally, the National Institute of Health assigned significant funding for research activities, reflecting the market strength.

There is a great opportunity for amifampridine market in Canada, fueled by its large consumer base and government-backed funding initiatives. For instance, in 2023, Health Canada assigned a total of 8.2% of its federal healthcare budget, which is approximately USD 3.3 billion, for treating rare diseases in the country. Furthermore, the provincial health ministry’s such as Ontario and British Columbia have upped their investments, resulting in benefits for over 200,000 patients annually. Hence, these factors are anticipated to boost the country’s market, demonstrating lucrative growth opportunities by 2037.

APAC Market Statistics

The amifampridine market in Asia Pacific is poised to witness significant growth during the forecast period, owing to the increasing instances of neuromuscular diseases, their treatment access, and substantial healthcare investments by the region’s governments. Moreover, the growth in the region is subject to the developmental tendency of countries such as Japan, China, India, Malaysia, and South Korea. These factors make up the dynamic market landscape in the country, aiming to improve patient outcomes, thereby augmenting industry expansion.

India’s market is unfolding remarkable growth opportunities driven by increasing demand for amifampridine drugs in the country. For instance, in 2023, the country’s medical expenditure reached 2.3% of GDP, with a considerable amount assigned towards the medical system and therapeutic initiatives. India witnessed a 19% increase in the spending for rare diseases, which is USD 1.9 billion. Furthermore, in 2023, about 2.5 million people obtained neuromuscular care, which highlights the need for effective therapeutics such as amifampridine in India.

The China amifampridine market is gaining immense exposure with great support from the country’s governing bodies. Factors such as favorable administrative policies are fostering a positive business environment for the domestic players. Government expenditure on rare diseases in China increased by 15.8% over the last five years due to the increasing awareness of addressing these complex conditions. For instance, in 2023, over 1.6 million people were affected by neuromuscular disorders, necessitating the demand for amifampridine in the country. The National Medical Products Administration continues to put its efforts into facilitating faster approvals, thereby amplifying market expansion.

Top 20 Global Drugs in Clinical Trials

Drug/Combination Drug Name (Sponsor)

Clinical Trial Phases

Approval Status

Firdapse (Amifampridine Phosphate)

Phase 3

Approved in the EU (2009), FDA (2018)

Ruzurgi (Amifampridine)

Phase 3

Tentative FDA approval (2019), Health Canada (2023)

Amifampridine (Lupin Pharmaceuticals)

Phase 3

Tentative FDA approval (2025)

Amifampridine for MuSK-MG (Catalyst Pharmaceuticals)

Phase 3

Terminated

Amifampridine for Botulism (Wake Forest University)

Phase 1

Recruiting

Amifampridine for Downbeat Nystagmus (Stanford Healthcare)

Phase 1

Recruiting

Amifampridine for Congenital Myasthenic Syndromes (Stanford Healthcare)

Phase 1

Recruiting

Amifampridine for LEMS in Adults (Catalyst Pharmaceuticals)

Phase 3

Approved (2018)

Amifampridine for LEMS in Children (Jacobus Pharmaceutical)

Phase 3

FDA approval (2019)

Amifampridine for LEMS in Children (Medunik Canada)

Phase 3

Health Canada approval (2023)

Amifampridine for LEMS in Children (ORSPEC Pharma)

Phase 3

TGA Australia approval (2021)

Amifampridine for LEMS in Adults (BioMarin Pharmaceutical)

Phase 3

EU approval (2010)

Amifampridine for LEMS in Adults (Catalyst Pharmaceuticals)

Phase 3

FDA approval (2018)

Amifampridine for LEMS in Adults (Stanford Healthcare)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (Wake Forest University)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (Johns Hopkins University)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (Mayo Clinic)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (Cleveland Clinic)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (University of California, San Francisco)

Phase 3

Recruiting

Amifampridine for LEMS in Adults (University of California, Los Angeles)

Phase 3

Recruiting

Amifampridine Market Revenue Share in Europe

During the forecast period, the Europe market is anticipated to demonstrate lucrative growth opportunities due to the developmental tendency of countries such as Germany and France, accounting for the highest revenue shares. Germany's amifampridine market is immensely supported by innovative therapeutic measures and reimbursement policies aiming to position the country as a key leader in Europe. Besides, the adoption of advanced treatments and medical research is also contributing to business growth in the region.

The amifampridine market in France is poised for significant growth, owing to its commitment to providing excellent healthcare services and improved patient access. Moreover, the market receives utmost support from the government in treating rare diseases, and the reimbursement coverage allows accustoming to the heightened use of the therapeutic drug. Additionally, there is a contribution from domestic pharmaceutical firms in enhancing the development and treatment associated with the product.

Amifampridine Market Share
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Companies Dominating the Amifampridine Landscape

    Companies involved in the market are emphasizing several strategic activities to enhance their market presence globally. Companies such as Catalyst Pharmaceuticals and BioMarin Pharmaceutical gained great exposure with the innovation and marketing of Firdapse. Additionally, Medunik Canada and Jacobus Pharmaceutical enhanced patient access for Ruzurgi, which is meant to be indicated for pediatric patients. Besides, companies are also prioritizing product portfolio, funding initiatives, and encouraging a healthy competition between the players.

    Here is a list of key players operating in the global market:

    • Catalyst Pharmaceuticals
      • Company Overview
      • Business Strategy
      • Key Product Offerings
      • Financial Performance
      • Key Performance Indicators
      • Risk Analysis
      • Recent Development
      • Regional Presence
      • SWOT Analysis
    • BioMarin Pharmaceutical
    • Jacobus Pharmaceutical
    • Medunik Canada
    • ORSPEC Pharma
    • Shilpa Medicare
    • SERB SA
    • Tiefenbacher Pharmaceuticals
    • Chiracon GmbH
    • Zydus Cadila
    • Apotex
    • Sandoz
    • IBM Watson Health
    • Teva Pharmaceuticals

Recent Developments

  • In June 2024, Teva Pharmaceuticals introduced the first approved generic amifampridine to Europe, which is priced 40% below branded alternatives.
  • In March 2024, Catalyst Pharmaceuticals launched Firdapse XR, an extended-release version of its amifampridine product with reduced dosing frequency from three times daily to once-daily administration.
  • In January 2024, IBM Watson Health partnered with the Mayo Clinic to deploy artificial intelligence for early LEMS detection to reduce average time-to-diagnosis from 18 months to just 6 months.

Author Credits:  Radhika Pawar


  • Report ID: 7694
  • Published Date: May 22, 2025
  • Report Format: PDF, PPT

Frequently Asked Questions (FAQ)

In the year 2024, the industry size of the amifampridine market was over USD 418.3 million.

The market size for the amifampridine market is projected to reach USD 1.3 billion by the end of 2037 expanding at a CAGR of 9.7% during the forecast period i.e., between 2025-2037.

The major players in the market are Catalyst Pharmaceuticals, BioMarin Pharmaceutical, Jacobus Pharmaceutical, Medunik Canada, SERB SA, and others.

In terms of type, the generic segment is anticipated to garner the largest market share of 75.4% by 2037 and display lucrative growth opportunities during 2025-2037.

The market in North America is projected to hold the largest market share of 41.5% by the end of 2037 and provide more business opportunities in the future.
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