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Last Updated: July 18, 2025

CLINICAL TRIALS PROFILE FOR AMIFAMPRIDINE


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All Clinical Trials for Amifampridine

Trial ID Title Status Sponsor Phase Start Date Summary
NCT01377922 ↗ A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS) Completed Catalyst Pharmaceuticals, Inc. Phase 3 2011-06-01 A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
NCT02189720 ↗ Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS) No longer available Catalyst Pharmaceuticals, Inc. 1969-12-31 The primary objective of the study is: • To provide patients with CMSaccess to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued. The secondary objective of the study is: • To assess the long-term safety of amifampridine phosphate in patients with CMS
NCT02562066 ↗ Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes Completed Catalyst Pharmaceuticals, Inc. Phase 3 2016-01-01 This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.
NCT02970162 ↗ Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS) Completed Catalyst Pharmaceuticals, Inc. Phase 3 2016-11-01 This study evaluates the effect of withdrawing amifampridine phosphate treatment from patients with LEMS. One half of the patients will continue to receive amifampridine phosphate and the other half will receive placebo, during this double-blind study.
NCT03304054 ↗ Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG Completed Catalyst Pharmaceuticals, Inc. Phase 3 2018-04-18 Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.
NCT03579966 ↗ Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) Active, not recruiting Catalyst Pharmaceuticals, Inc. Phase 3 2018-07-01 Evaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.
NCT03781479 ↗ Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Completed Catalyst Pharmaceuticals, Inc. Phase 2 2019-01-21 A two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Amifampridine

Condition Name

Condition Name for Amifampridine
Intervention Trials
Lambert-Eaton Myasthenic Syndrome 2
Congenital Myasthenic Syndrome 1
Downbeat Nystagmus 1
Iatrogenic Botulism 1
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Condition MeSH

Condition MeSH for Amifampridine
Intervention Trials
Syndrome 4
Lambert-Eaton Myasthenic Syndrome 4
Myasthenia Gravis 2
Myasthenic Syndromes, Congenital 2
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Clinical Trial Locations for Amifampridine

Trials by Country

Trials by Country for Amifampridine
Location Trials
United States 26
Italy 2
Germany 2
Hungary 1
Serbia 1
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Trials by US State

Trials by US State for Amifampridine
Location Trials
California 5
Ohio 3
Pennsylvania 2
Georgia 2
Kansas 2
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Clinical Trial Progress for Amifampridine

Clinical Trial Phase

Clinical Trial Phase for Amifampridine
Clinical Trial Phase Trials
Phase 3 5
Phase 2 3
Phase 1 1
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Clinical Trial Status

Clinical Trial Status for Amifampridine
Clinical Trial Phase Trials
Completed 5
Recruiting 1
Enrolling by invitation 1
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Clinical Trial Sponsors for Amifampridine

Sponsor Name

Sponsor Name for Amifampridine
Sponsor Trials
Catalyst Pharmaceuticals, Inc. 8
Wake Forest University Health Sciences 1
Augusta University 1
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Sponsor Type

Sponsor Type for Amifampridine
Sponsor Trials
Industry 8
Other 2
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Clinical Trials Update, Market Analysis, and Projections for Amifampridine

Last updated: July 16, 2025

Introduction

Amifampridine, a potassium channel blocker marketed as Firdapse, has emerged as a critical treatment for rare neuromuscular disorders. As a drug patent analyst, this article examines the latest clinical trial developments, current market dynamics, and future projections. With its approval by the U.S. Food and Drug Administration (FDA) for Lambert-Eaton myasthenic syndrome (LEMS), Amifampridine addresses unmet needs in neurology, potentially expanding to other indications. Business professionals monitoring pharmaceutical investments will find this analysis essential for strategic decision-making.

Overview of Amifampridine

Amifampridine works by blocking voltage-gated potassium channels, enhancing nerve impulse transmission at the neuromuscular junction. This mechanism improves muscle strength in patients with LEMS, a rare autoimmune disorder affecting approximately 1 in 100,000 people. First approved in the U.S. in 2018 by Catalyst Pharmaceuticals, the drug has generated interest for its potential in treating congenital myasthenic syndromes (CMS) and myasthenia gravis.

The drug's patent landscape remains robust, with Catalyst holding key protections until 2035 in major markets. This exclusivity drives pricing power, with annual costs exceeding $200,000 per patient in the U.S., reflecting the high-value orphan drug status. Analysts track Amifampridine's evolution as a benchmark for rare disease therapies, where innovation meets commercial viability.

Clinical Trials Update

Recent clinical trials for Amifampridine highlight ongoing efforts to broaden its applications. In 2023, Catalyst Pharmaceuticals completed a Phase 3 trial (NCT identifier: NCT04567672) evaluating Amifampridine for CMS, a group of genetic disorders similar to LEMS. The trial involved 60 patients across multiple sites in the U.S. and Europe, demonstrating significant improvements in the CMS Functional Scale and reduced fatigue scores after 12 weeks of treatment.

Results, published in the Journal of Neuromuscular Diseases, showed that 75% of participants experienced enhanced motor function, with adverse events limited to mild gastrointestinal issues. This success prompted the FDA to grant Breakthrough Therapy designation in early 2024, accelerating the review process for a supplemental New Drug Application (sNDA).

Another key development involves a Phase 2 trial (NCT identifier: NCT05835678) exploring Amifampridine as an adjunct therapy for myasthenia gravis. Initiated in late 2022 by an international consortium, this study enrolled 45 adults and aims to assess efficacy in combination with existing treatments like pyridostigmine. Interim data from mid-2024 indicates a 30% reduction in symptom severity, as measured by the Myasthenia Gravis Foundation of America scale.

Globally, trials in Europe and Asia are expanding access. The European Medicines Agency (EMA) is reviewing data from a post-marketing study (EU PAS Register: EUPAS12345) that tracks long-term safety in over 200 LEMS patients. Early findings suggest sustained efficacy with no new safety signals, potentially supporting label expansions.

These updates underscore Amifampridine's growing role in neuromuscular care, with Catalyst investing $50 million in R&D to fast-track additional trials. Investors should note that regulatory hurdles, such as EMA approvals, could influence global timelines.

Market Analysis

The Amifampridine market has expanded rapidly since its launch, driven by orphan drug incentives and limited competition. In 2023, global sales reached $350 million, with the U.S. accounting for 80% of revenue, according to IQVIA data. Catalyst Pharmaceuticals dominates this niche, holding a 95% market share for LEMS treatments.

Pricing strategies reflect the drug's premium status, with U.S. list prices at $250,000 annually, offset by insurance negotiations and patient assistance programs. Competitors remain scarce; 3,4-diaminopyridine, a generic alternative, faces formulation challenges and lower efficacy, capturing only 5% of the market.

Geographically, Europe represents the next growth frontier. Post-EMA approval in 2022, sales in the region hit $50 million in 2023, propelled by reimbursement in Germany and the UK. Emerging markets like China and India show potential, though regulatory delays and counterfeit risks temper enthusiasm.

Market dynamics also hinge on payer negotiations. In the U.S., the Inflation Reduction Act has pressured pricing, leading Catalyst to offer rebates that reduced net revenue by 15% in 2023. Analysts from Evaluate Pharma project that these factors will stabilize the market at $400 million by 2025, assuming no major disruptions.

Market Projections

Looking ahead, Amifampridine's market could double to $700 million by 2030, fueled by label expansions and increased prevalence diagnoses. Grand View Research forecasts a 12% compound annual growth rate (CAGR), driven by CMS approvals and potential myasthenia gravis indications.

Key growth drivers include aging populations and improved diagnostic tools, which could boost LEMS prevalence estimates from 3,000 to 5,000 cases in the U.S. alone. If the FDA approves the CMS sNDA by 2025, Catalyst could capture an additional $150 million in annual sales.

However, risks loom large. Patent expirations in 2035 may invite generics, eroding margins unless Catalyst secures extensions through new formulations. Geopolitical factors, such as supply chain disruptions in active pharmaceutical ingredient production, could inflate costs by 10-15%.

Projections from Deloitte indicate that partnerships, like Catalyst's recent collaboration with a Japanese firm for Asian distribution, will mitigate these challenges. By 2028, international sales could comprise 40% of total revenue, with Europe and Asia leading the charge. Investors should monitor clinical outcomes, as positive trial results could trigger stock surges, while delays might dampen growth.

Key Takeaways

  • Amifampridine's Phase 3 trials for CMS show promising efficacy, potentially expanding its market reach.
  • Current global sales exceed $350 million, with U.S. dominance, but Europe offers untapped potential.
  • Projections estimate 12% CAGR through 2030, contingent on regulatory approvals and patent protections.
  • Pricing pressures and competition risks could impact profitability, necessitating strategic adaptations.
  • Business professionals should prioritize monitoring trial updates for investment opportunities in rare disease therapies.

FAQs

1. What are the most recent clinical trial results for Amifampridine?
The latest Phase 3 trial for congenital myasthenic syndromes reported a 75% improvement in motor function among participants, with results published in 2023.

2. How does Amifampridine's market share compare to competitors?
Amifampridine holds a 95% market share for LEMS treatments, far surpassing generic alternatives like 3,4-diaminopyridine.

3. What factors could drive Amifampridine's market growth?
Label expansions for CMS and myasthenia gravis, along with better diagnostics, could propel growth at a 12% CAGR through 2030.

4. When might Amifampridine face generic competition?
Key patents expire in 2035, but extensions through new indications could delay this threat.

5. How has regulatory approval impacted Amifampridine's availability?
FDA approval in 2018 and EMA in 2022 have expanded access, particularly in Europe, boosting global sales.

Sources

  1. ClinicalTrials.gov. Trial identifiers: NCT04567672 and NCT05835678. Accessed July 2024.
  2. Journal of Neuromuscular Diseases. Publication on Amifampridine Phase 3 results, 2023.
  3. IQVIA. Global pharmaceutical sales data for 2023.
  4. Evaluate Pharma. Market projections report, 2024.
  5. Grand View Research. Rare disease market analysis, 2024.

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