Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing medicines for rare diseases in the United States. Its portfolio includes Firdapse, Fycompa, and AGAMREE, with additional partnered programs.
The Consolidated Appropriations Act, 2026, enacted February 3, reauthorizes the rare pediatric disease priority review voucher program through 2029, clarifies orphan drug exclusivity scope, and mandates increased transparency in FDA's generic drug determinations.
The Consolidated Appropriations Act, 2026 enacted February 3 narrows orphan drug exclusivity to specific approved uses rather than entire disease categories, overriding a 2021 court decision and codifying FDA's longstanding interpretation.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06564974 |
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT |
RECRUITING | |
| NCT03819660 |
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 |
TERMINATED | PHASE2 |
| NCT03781479 |
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients |
COMPLETED | PHASE2 |
| NCT03579966 |
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) |
TERMINATED | PHASE3 |
| NCT03304054 |
Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG |
COMPLETED | PHASE3 |
| NCT03062631 |
Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia |
NO_LONGER_AVAILABLE | |
| NCT02970162 |
Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS) |
COMPLETED | PHASE3 |
| NCT02562066 |
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes |
COMPLETED | PHASE3 |
| NCT02189720 |
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome |
APPROVED_FOR_MARKETING | |
| NCT01493596 |
A Safety, Tolerability and Pharmacokinetic Study of CPP-115 |
COMPLETED | PHASE1 |
