Firdapse

(Amifampridine Phosphate)

PreclinicalSmall molecule

CCatalyst Pharmaceuticals, Inc.

Firdapse is one of 5 small molecule programs targeting Congenital Myasthenic Syndrome. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Congenital Myasthenic SyndromeActive, not recruiting

Congenital Myasthenic SyndromePreclinical

Active, not recruiting

Competition

Competitive Landscape

4 competitors in Congenital Myasthenic Syndrome

#DrugCompanyPhaseTrials

Ephedrine Sulfate

DDr. Reddys Laboratories, SA

Adimanebart IV

1 3

Alloex

TThe Foundation for Orthopaedics and Regenerative Medicine

Phase 1

1 4

ARGX-119

Argenx

Phase 1

1 5

Firdapse

CCatalyst Pharmaceuticals, Inc.

Preclinical

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

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

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

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

Congenital myasthenic syndromes1 trial

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

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

2 more indications

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

MPR1 trial

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

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

Regulatory

FDA Approval Timeline[4]

Nov 28, 2018

FIRDAPSE

NDA NDA208078

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]
OpenFDA — FDA approval records (NDA NDA208078). U.S. Food & Drug Administration.
[5]
SEC EDGAR — Catalyst Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

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

Firdapse

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Spinal Muscular Atrophy2 trials

Congenital Myasthenic Syndrome1 trial

Efficacy1 trial

Myasthenia Gravis1 trial

Congenital myasthenic syndromes1 trial

Lambert Eaton Myasthenic Syndrome1 trial

MPR1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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