amifampridine (Firdapse)

following a full submission:

amifampridine phosphate (Firdapse®) is not recommended for use within NHS Scotland.

Indication under review: Symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults.

There are no clinical data for amifampridine phosphate and efficacy has been extrapolated from studies of amifampridine base (3,4-diaminopyridine), to which amifampridine phosphate has been accepted to be bioequivalent by the European Medicines Agency.  In randomised controlled studies in patients with LEMS, 3,4-diaminopyridine treatment was associated with greater improvement in muscle strength and neuromuscular transmission than placebo.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition, the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.