CALL TOLL FREE 1-844-347-3277 OR EMAIL FOR MORE INFORMATION
Welcome to the amifampridine phosphate Expanded Access Program (EAP). Amifampridine phosphate is an investigational treatment that may be available at no cost to patients diagnosed with the neuromuscular disorders Lambert-Eaton Myasthenic Syndrome (LEMS) or certain types of Congenital Myasthenic Syndromes (CMS).
The Expanded Access Program is designed to provide access to treatment for patients diagnosed with LEMS or certain types of CMS when the treating physician feels that amifampridine phosphate may help. The investigational medication is only available through the EAP under the direction and care of a patient’s treating physician.
The FDA has not yet approved amifampridine phosphate for the treatment of either LEMS or CMS.
AMIFAMPRIDINE PHOSPHATE DETAILS AND EAP ELIGIBILITY CRITERIA
- Demonstrated positive results in one Phase 3 clinical study for LEMS
- Granted FDA Orphan Drug designation for LEMS and CMS
- Designated as a “Breakthrough Therapy” by the FDA for its review of the product for LEMS as a potentially substantial improvement over existing therapies
- Novel phosphate salt formulation allows for storage at room temperature
- Amifampridine phosphate is produced utilizing Good Manufacturing Practices (GMP) which ensure product quality and consistency
- Approved and marketed for more than 5 years in the EU for the treatment of LEMS
The most common adverse events in a Phase 3 clinical trial for this investigational treatment with LEMS patients were oral and digital paresthesias (eg, tingling) nausea, and headache
PATIENT ELIGIBLITY CRITERIA
- Patients must be ≥2 years of age
- Must have a confirmed diagnosis of LEMS or documentation of certain types of CMS
- History of epilepsy
- Known active brain metastasis
- Risk factors for Torsades de pointes
- QTcB > 450 ms (males) or 470 ms (females)
- Hepatic impairment
– AST, ALT, or total bilirubin > 1.5 x upper limit of normal
- Creatinine clearance <30 mL/min