Amifampridine is used predominantly to treat a range of rare muscle diseases. its free base
form has been in use to treat congenital myasthenic syndromes and Lambert–Eaton
myasthenic syndrome (LEMS) on compassionate use programs since the 1990s. However, it
was not recommended as a first line treatment for LEMS till 2006. Amifampridine is in some
cases also used in the treatment of psoriasis.
Congenital myasthenic syndromes are treated using Amifampridine. Especially in those with
depleted choline acetyltransferase, downstream kinase 7. It is also involved when defects
causes "fast channel" behavior of the acetylcholine receptor.
Amifampridine is sold under the Brand names Firdapse and Zenas.
Around 2000 doctors in Paris created a phosphate salt, further developed through a series of
companies. Finally, in 2009, BioMarin Pharmaceutical got approval in EU under the trade
name Firdapse. Later in 2012, Catalyst Pharmaceuticals bought licensed to sell in the US.
As for 2018, It has been approved by the US FDA for treating adults with LEMS. The FDA
granted the approval to Jacobus Pharmaceutical, which has the only other treatment
approved for LEMS in adults.
Dosage and administration
Amifampridine is sold as tablets of 10mg strength. In the treatment of Lambert-Eaton
Myasthenic Syndrome, the recommended adult dose is 15 to 30 mg, given every day in 3 to
5 divided doses initially. This dose may be increased by 5 mg per day with maximum daily
dose not to exceed 80 mg.
The cost of Amifampridine
As of 2018, in Europe, Amifampridine phosphate cost $60,000 for a treatment lasting a full
year. In the USA, consumers have to shell out an average retail price of $23,603 for a supply
of 120 tablets. In the global ingredients market, high grade Amifampridine cost USD 25,000
for a kilogram.
How does Amifampridine Work?
As symptomatic treatment, Amifampridine increases acetylcholine concentrations at the
neuromuscular junction. Its actions block presynaptic fast voltage-gated potassium channels,
which results in prolongation of cell membrane depolarization and action potential. It also
augments the amount if calcium being transported into the nerve endings, which enhances
the exocytosis of acetylcholine-containing vesicles. Amifampridine actions also enhance
impulse transmission at central, autonomic, and neuromuscular synapses. Amifampridine
increases the strength in our muscles. It also increases potential amplitudes in complex
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