Product Description
Amifampridine is used to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disease, wherein the immune system attacks the connection between the nerve and muscle (neuromuscular junction) and blocks the ability of the nerve cells to send signals to the muscle cells, leading to muscle weakness of the arms or legs. (Sourced from: https://www.mayoclinic.org/drugs-supplements/amifampridine-oral-route/description/drg-20463409)
Mechanisms of Action: Potassium Channel Blocker
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Orphan Drug - Lambert-Eaton Myasthenic Syndrome *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Canada | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Latvia | Lithuania | Netherlands | Norway | Poland | Portugal | Slovakia | Slovenia | Spain | Sweden | Switzerland | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: Catalyst
Company Location: Eastern America
Company CEO: Nassim Usman
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Japan, Unknown Location
Active Clinical Trial Count: 2
Recent & Upcoming Milestones
- PDUFA target action date of June 4, 2024 for increasing Firdapse's daily dose to 100mg for Lambert-Eaton Myasthenic Syndrome.
Highest Development Phases
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
jRCT2031220702 |
jRCT2031220702 | P3 |
Recruiting |
Unknown |
2024-12-31 |
|||
jRCT2031210552 |
jRCT2031210552 | P3 |
Active, not recruiting |
Unknown |
2024-03-31 |