- The first patient has been enrolled in the second Phase 3 study, LMS-003, assessing Catalyst Pharmaceuticals' (NASDAQ:CPRX) Firdapse (amifampridine phosphate) for the treatment Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder characterized by muscle weakness in the limbs.
- LMS-003 will enroll ~28 subjects and will have the same co-primary endpoints as the first Phase 3 trial. Top-line data and the resubmission of a New Drug Application (NDA) to the FDA should happen in H2 2017.
- The FDA issued a "Refusal to File" letter in February in response to the company's first NDA, which meant that the filing was incomplete and required additional supporting data.
- Amifampridine enhances neuromuscular transmission by blocking the potassium channel. This causes depolarization of the presynaptic membranes which opens the calcium channel thereby enabling muscle fibers to contract.
- Previously: FDA rejects Catalyst Pharma's Firdapse NDA; shares slump 38% (Feb. 17)
Enrollment underway in Catalyst Pharma's second late-stage study of Firdapse in LEMS
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