| dbo:abstract | - Amifampridine 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 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 trade name Firdapse, and which licensed the US rights to Catalyst Pharmaceuticals in 2012. As of January 2017, Catalyst and another US company, , which had been manufacturing and giving it away for free since the 1990s, were both seeking FDA approval for their iterations and marketing rights. Amifampridine phosphate has orphan drug status in the EU for Lambert–Eaton myasthenic syndrome and Catalyst holds both an orphan designation and a breakthrough therapy designation in the US. In May 2019 the U.S. Food and Drug Administration (FDA) approved amifampridine tablets under the trade name Ruzurgi for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA approval of a treatment specifically for pediatric patients with LEMS. The FDA granted the approval of Ruzurgi to Jacobus Pharmaceutical. The only other treatment approved for LEMS (Firdapse) is only approved for use in adults. (en)
- Amifampridin ist ein Arzneistoff aus der Gruppe der reversiblen Kaliumkanal-Blocker, der u. a. zur Behandlung der Muskelschwäche beim myasthenischen Lambert-Eaton-Syndroms (LEMS) eingesetzt wird. Chemisch ist der Stoff den Aminopyridinen zuzuordnen. (de)
- L'amifampridine ou 3,4-diaminopyridine est un composé organique dérivé de la pyridine par substitution des carbones 3 et 4 par des groupes amine. Elle a pour effet de bloquer les canaux potassiques voltage-dépendants. Elle est utilisée comme médicament chez l'homme, sous forme de phosphate d'amifampridine, dans la prise en charge du syndrome myasthénique de Lambert-Eaton. (fr)
- 3,4-diaminopyridine is een aromatisch diamine afgeleid van pyridine. Het EG-nummer is 200-220-9. (nl)
- 3,4-Diaminopiridina (o 3,4-DAP) è un composto organico con la formula C5H3 N(NH2)2. Esso è formalmente derivato dalla piridina per sostituzione nelle posizioni 3 e 4 con un gruppo amminico. Con il nome di amifampridina secondo la denominazione comune internazionale (INN), esso è usato soprattutto come farmaco per il trattamento di alcune malattie muscolari rare. In Europa, il sale fosfato dell'amifampridina è stato concesso in licenza come Firdapse alla, nel 2010, come farmaco orfano. (it)
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| dbp:drugbank | - DB11640 (en)
- DBSALT002818 (en)
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| dbp:iupacName | - Pyridine-3,4-diamine (en)
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| dbp:metabolism | - Acetylation to 3-N-acetyl­amifampridine (en)
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| dbp:stdinchikey | - OYTKINVCDFNREN-UHFFFAOYSA-N (en)
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| dbp:synonyms | - pyridine-3,4-diamine, 3,4-diaminopyridine, 3,4-DAP (en)
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- RU4S6E2G0J (en)
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| rdfs:comment | - Amifampridin ist ein Arzneistoff aus der Gruppe der reversiblen Kaliumkanal-Blocker, der u. a. zur Behandlung der Muskelschwäche beim myasthenischen Lambert-Eaton-Syndroms (LEMS) eingesetzt wird. Chemisch ist der Stoff den Aminopyridinen zuzuordnen. (de)
- L'amifampridine ou 3,4-diaminopyridine est un composé organique dérivé de la pyridine par substitution des carbones 3 et 4 par des groupes amine. Elle a pour effet de bloquer les canaux potassiques voltage-dépendants. Elle est utilisée comme médicament chez l'homme, sous forme de phosphate d'amifampridine, dans la prise en charge du syndrome myasthénique de Lambert-Eaton. (fr)
- 3,4-diaminopyridine is een aromatisch diamine afgeleid van pyridine. Het EG-nummer is 200-220-9. (nl)
- 3,4-Diaminopiridina (o 3,4-DAP) è un composto organico con la formula C5H3 N(NH2)2. Esso è formalmente derivato dalla piridina per sostituzione nelle posizioni 3 e 4 con un gruppo amminico. Con il nome di amifampridina secondo la denominazione comune internazionale (INN), esso è usato soprattutto come farmaco per il trattamento di alcune malattie muscolari rare. In Europa, il sale fosfato dell'amifampridina è stato concesso in licenza come Firdapse alla, nel 2010, come farmaco orfano. (it)
- Amifampridine 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 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. (en)
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| rdfs:label | - Amifampridine (en)
- Amifampridin (de)
- Amifampridine (fr)
- 3,4-Diaminopiridina (it)
- 3,4-diaminopyridine (nl)
- 3,4-二氨基吡啶 (zh)
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