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amifampridine
EntityQ411707· pop 16· linked from 351 articles

amifampridine

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Also known as 3,4-DAP, 3,4-diaminopyridine, Firdapse®, Zenas®

Amifampridine phosphate is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and approved by the FDA for Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc forms of the drug, since there was no marketed form.

Key facts

Drug.Verifiedfields
changed
Drug.Watchedfields
changed
Drug.verifiedrevid
477217342
Drug.image
Diaminopyridine.png
Drug.image_class
skin-invert-image
Drug.width
120
Drug.image2
3,4-diaminopyridine-3D-balls.png
Drug.image_class2
bg-transparent
Drug.tradename
Firdapse, Ruzurgi
Drug.DailyMedID
Amifampridine
Drug.pregnancy_AU
C
Drug.routes_of_administration
By mouth
Drug.ATC_prefix
N07
Drug.ATC_suffix
XX05
Drug.legal_AU
S4
Drug.legal_CA
Rx-only
Drug.legal_UK
POM
Drug.legal_US
Rx-only

via Wikipedia infobox

~13 min read

Encyclopedic overview

11 sections
Contents
  • Medical uses
  • Contraindications
  • Adverse effects
  • Interactions
  • Pharmacology
  • Mechanism of action
  • Pharmacokinetics
  • Chemistry
  • History
  • Research
  • References

Amifampridine phosphate is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and approved by the FDA for Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc forms of the drug, since there was no marketed form.

Around 2000 doctors at Assistance Publique – Hôpitaux de Paris created a phosphate salt form, which was developed through a series of companies ending with BioMarin Pharmaceutical which obtained European approval in 2009 under the brand name Firdapse, and which licensed the US rights to Catalyst Pharmaceuticals in 2012. As of January 2017, Catalyst and another US company, Jacobus Pharmaceutical, which had been manufacturing and giving it away for free since the 1990s, were both seeking FDA approval for their iterations and marketing rights.

Excerpted from Wikipedia’s “amifampridine” article, available under the CC BY-SA 4.0 licence.