The Amifampridine Phosphate 
Expanded Access Program (EAP)

The Amifampridine Phosphate 
Expanded Access Program (EAP)

CALL 1-844-347-3277

CALL TOLL FREE 1-844-347-3277 OR EMAIL FOR MORE INFORMATION

The investigational treatment amifampridine phosphate 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)

Expanded Access Programs are intended to give patients access to investigational medications for serious diseases or conditions when there is no comparable or satisfactory treatment available. Participating physicians are able to provide the medication to their eligible U.S. patients while it is still under development. For more information go to: clinicaltrials.gov

The FDA has not yet approved amifampridine phosphate for the treatment of either LEMS or CMS

ABOUT AMIFAMPRIDINE PHOSPHATE

Amifampridine phosphate is an investigational drug that has demonstrated positive results for the treatment for LEMS in a recent global Phase 3 clinical trial. The FDA has granted it an Orphan Drug designation for the neuromuscular disorders LEMS and CMS. This novel phosphate salt formulation allows for storage at room temperature and is manufactured under Good Manufacturing Practices (GMP) to ensure product quality and consistency.

Amifampridine phosphate may be available at no cost for patients diagnosed with Lambert-Eaton Myasthenic Syndrome (LEMS) or certain types of Congenital Myasthenic Syndromes (CMS) through this EAP.

Amifampridine phosphate has been approved and marketed for more than 5 years in the European Union for the treatment of LEMS. The FDA has not yet approved amifampridine phosphate for the symptomatic treatment of either LEMS or CMS.

Catalyst Pharmaceuticals is committed to ensuring all eligible patients access to treatment.

No serious side effects attributable to amifampridine phosphate were reported during the Phase 3 trial. The most common side effects were oral and digital paresthesias (e.g. tingling), nausea, and headache.

Reference: 1. Data on file, Phase III Clinical Trial. 2014. Catalyst Pharmaceuticals, Inc.

ABOUT THE AMIFAMPRIDINE PHOSPHATE EXPANDED ACCESS PROGRAM (EAP)

Catalyst Pharmaceuticals introduced the amifampridine phosphate EAP to help patients who have been diagnosed with LEMS or certain types of CMS gain access to an investigational medication at a time when there are no FDA-approved treatments for these rare, debilitating neuromuscular diseases.

This expanded access program will allow healthcare providers to prescribe individually determined doses of amifampridine phosphate to eligible patients. It is also designed to collect long-term safety data through a variety of exams and laboratory tests performed as standard of care by treating physicians.

Catalyst Pharmaceuticals is committed to ensuring all eligible patients have access to treatment. The FDA has not yet approved amifampridine phosphate for the symptomatic treatment of either LEMS or CMS.

Call toll free 1-844-347-3277 or submit your email here if you are interesting in getting more information about the EAP enrollment qualifications and the study protocol

PATIENT ELIGIBILITY CRITERIA

Patients who are already taking amifampridine base, as well at those not currently taking amifampridine, may participate in the program.

Minimum inclusion criteria for participation include:

– Patients must be ≥2 years of age

– Confirmed diagnosis of LEMS or documentation of certain types of CMS

Amifampridine phosphate may have an affect on the following CMS genetic mutations: acetylcholine receptor defect, Rapsyn deficiency, MuSK deficiency, Dok-7 deficiency, SYT2 mutations, SNAP25B deficiency, and fast channel syndrome.

PATIENT EXCLUSION CRITERIA

The exclusion criteria for the Expanded Access Program (EAP) are as follows:

  • 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

PLEASE CLICK HERE TO DOWNLOAD AN INFORMATIONAL BROCHURE ON
THE AMIFAMPRIDINE PHOSPHATE EAP, LEMS AND CMS