Amifampridine Phosphate

Phase 3Small molecule

Amifampridine Phosphate is one of 77 small molecule programs targeting Congenital myasthenic syndromes. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Congenital Myasthenic SyndromesActive, not recruiting

Congenital Myasthenic SyndromeActive, not recruiting

Myasthenia GravisActive, not recruiting

Spinal Muscular AtrophyActive, not recruiting

Congenital Myasthenic SyndromesPhase 3

Active, not recruiting

Congenital Myasthenic SyndromePreclinical

Active, not recruiting

Myasthenia GravisPhase 3

Active, not recruiting

Spinal Muscular AtrophyPhase 2

Active, not recruiting

Competition

Competitive Landscape

102 competitors in Myasthenia Gravis

#DrugCompanyPhaseTrials

Rituxan

Roche

Phase 3

7 2

Ultomiris

AstraZeneca

Phase 3

5 3

Vyvgart

Argenx

Phase 3

17 4

Soliris

AstraZeneca

Phase 3

4 5

Zilbrysq

UCB

Phase 3

4 6

Cellcept

Roche

Phase 3

4 7

Fabhalta

Johnson & Johnson

Phase 3

3 8

Arava

Sanofi

Phase 3

1 9

Rhapsido

Novartis

Phase 3

1 10

ENSPRYNG

Roche

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy2 trials

Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

Completed·CCatalyst Pharmaceuticals, Inc.·Phase 2·NCT03781479·13 enrolled

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

Terminated·CCatalyst Pharmaceuticals, Inc.·Phase 2·NCT03819660·13 enrolled

Myasthenia Gravis1 trial

Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)

Terminated·CCatalyst Pharmaceuticals, Inc.·Phase 3·NCT03579966·63 enrolled

Efficacy1 trial

Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)

Completed·CCatalyst Pharmaceuticals, Inc.·Phase 3·NCT02970162·26 enrolled

Congenital myasthenic syndromes1 trial

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

Completed·CCatalyst Pharmaceuticals, Inc.·Phase 3·NCT02562066·20 enrolled

Lambert Eaton Myasthenic Syndrome1 trial

A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)

Completed·CCatalyst Pharmaceuticals, Inc.·Phase 3·NCT01377922·38 enrolled

2 more indications

Congenital Myasthenic Syndrome1 trial

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

Approved for marketing·CCatalyst Pharmaceuticals, Inc.··NCT02189720

MPR1 trial

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

Completed·CCatalyst Pharmaceuticals, Inc.·Phase 3·NCT03304054·93 enrolled

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for Amifampridine Phosphate. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Amifampridine Phosphate compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Amifampridine Phosphate. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Catalyst Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: CCatalyst Pharmaceuticals, Inc.

Amifampridine Phosphate

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Spinal Muscular Atrophy2 trials

Myasthenia Gravis1 trial

Efficacy1 trial

Congenital myasthenic syndromes1 trial

Lambert Eaton Myasthenic Syndrome1 trial

Congenital Myasthenic Syndrome1 trial

MPR1 trial

References

Drug Info

Quick Facts

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