NCT02189720APPROVED_FOR_MARKETINGanonymous

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

Sponsor

Source record

Catalyst Pharmaceuticals, Inc.

Phase

Source record

Expanded Access

Modality

AI-normalized

small molecule

Target

AI-normalized

Amifampridine phosphate (3,4-Diaminopyridine Phosphate) acts as a potassium channel blocker, enhancing the release of acetylcholine at the neuromuscular junction, thereby improving neuromuscular transmission.

Indication / condition

AI-normalized

Congenital Myasthenic Syndrome

Intervention

Source record

Amifampridine Phosphate

Source & freshness

Source record

NCT ID

NCT02189720

Original source

ClinicalTrials.gov

Source last updated

Mar 26, 2025

Ingested at

Jun 17, 2026

Internal sync

Jun 17, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record

View original source fields

NCT ID

NCT02189720

Title

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

Sponsor

Catalyst Pharmaceuticals, Inc.

Status

APPROVED_FOR_MARKETING

Phase

Expanded Access

Condition raw

Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired

Condition normalized

Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired

Modality raw

small molecule

Modality normalized

small molecule

Target raw

Target normalized

Interventions

Amifampridine Phosphate

Public preview

Source record

Catalyst Pharmaceuticals, Inc. is leveraging the expanded access protocol for amifampridine phosphate to provide treatment for pediatric patients with Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS). The approval for marketing of Firdapse® for adult patients with LEMS in November 2018 and for pediatric patients aged 6 years and older in September 2022 positions the company favorably within the niche market of rare neuromuscular disorders. The ongoing expanded access study allows for the collection of long-term safety data, which may enhance the product's value proposition and support future regulatory submissions for additional indications. The competitive landscape includes other neuromuscular agents, but amifampridine's unique mechanism and established safety profile may provide a competitive edge.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 17, 2026

NCT ID

NCT02189720

Status

APPROVED_FOR_MARKETING

Phase

Expanded Access

Sponsor

Catalyst Pharmaceuticals, Inc.

Executive brief

Investment-Ready Snapshot

Source & freshness

Provenance

SourceClinicalTrials.gov

Source updatedMar 26, 2025

Internal syncJun 17, 2026

Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT02189720

Indication

Congenital Myasthenic Syndrome

Modality

small molecule

Target

Intervention

Amifampridine Phosphate

Source record