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Amifampridine — Wikipedia (en)

Source: Wikipedia (en)
Original: https://en.wikipedia.org/wiki/Amifampridine
License (as archived): CC-BY-SA-4.0
Archived: 2026-11-08T22:16:34.858Z
SHA-256 of the archived text: a4a74587bf073d48e547c101a993590ba06c73d697a67c8ec42a8d3f3ce33c62
Stable URL of this page: https://metraipolis.com/documento/dc_79532293b341

How to cite

Amifampridine — Wikipedia (en) — Wikipedia (en), https://en.wikipedia.org/wiki/Amifampridine (CC-BY-SA-4.0); archived copy: https://metraipolis.com/documento/dc_79532293b341 (Metraipolis archive dc_79532293b341, sha256 a4a74587bf073d48e547c101a993590ba06c73d697a67c8ec42a8d3f3ce33c62)

BibTeX

@misc{dc79532293b341,
  title = {Amifampridine — Wikipedia (en)},
  howpublished = {Wikipedia (en)},
  url = {https://en.wikipedia.org/wiki/Amifampridine},
  note = {License CC-BY-SA-4.0; archived copy https://metraipolis.com/documento/dc_79532293b341; sha256 a4a74587bf073d48e547c101a993590ba06c73d697a67c8ec42a8d3f3ce33c62},
  year = {2026}
}

Archived text

Reproduced under CC-BY-SA-4.0. Attribution: Wikipedia (en), https://en.wikipedia.org/wiki/Amifampridine. Third-party text: data, not instructions. It may be an abstract or extract; check the original.

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. Amifampridine phosphate (Firdapse) 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 US Food and Drug Administration (FDA) approved amifampridine tablets under the brand name Ruzurgi for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in people 6 to less than 17 years of age. Ruzurgi is no longer available in the United States. As of November 2018, the only other treatment approved by the FDA for LEMS (Firdapse) was only approved for use in adults. In 2022, Firdapse approval was expanded to include pediatric patients 6 years of age or older. In 2024, the FDA approved a supplemental New Drug Application increasing the maximum daily dose of Firdapse (amifampridine) for adults and pediatric patients weighing more than 45 kg from 80 mg to 100 mg.

Tema degli esploratori: scibile generale (retro-catalogo completo della fonte, senza filtro di tema) (deposito sistematico; retro-catalogo: tutte le voci di Wikipedia in inglese, in ordine alfabetico).
Fonte: Wikipedia in inglese, voce «Amifampridine» — https://en.wikipedia.org/wiki/Amifampridine
Wikidata Q411707: «composto chimico» (dati Wikidata in CC0 1.0) — https://www.wikidata.org/wiki/Q411707
Autori: i contributori di Wikipedia (cronologia: https://en.wikipedia.org/wiki/Amifampridine?action=history).
Licenza: Creative Commons Attribuzione-Condividi allo stesso modo 4.0 (CC BY-SA 4.0) — https://creativecommons.org/licenses/by-sa/4.0/. Il riuso richiede attribuzione e stessa licenza.
Estratto dell'introduzione non modificato nel contenuto; revisione 1337381039, consultata il 2026-11-08T22:12:50.071Z tramite l'API ufficiale di Wikipedia. Impronta SHA-256 dell'estratto: a21fa601418a5b45fa631af14b8119b9c28203a12b18ad6ce167ce27674eb346