🇪🇺 AVXS-101 in European Union

EMA authorised AVXS-101 on 18 May 2020

Marketing authorisation

EMA — authorised 18 May 2020

  • Application: EMEA/H/C/004750
  • Marketing authorisation holder: Novartis Europharm Limited
  • Local brand name: Zolgensma
  • Indication: Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
  • Pathway: conditional, orphan, ATMP, PRIME
  • Status: approved

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Other Rare Disease approved in European Union

Frequently asked questions

Is AVXS-101 approved in European Union?

Yes. EMA authorised it on 18 May 2020.

Who is the marketing authorisation holder for AVXS-101 in European Union?

Novartis Europharm Limited holds the EU marketing authorisation.