Limited Global Access to Zolgensma announced by Avexis

20 December 2019

Clinicians and SMA patient groups in SMA Europe have welcomed the news that Avexis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate. A statement will be coming shortly from SMA Europe (of which SMA UK are members).

Avexis have agreed to further discussions in the new year but are committed to the launch.

Currently it is not known if UK regulations will allow UK patients to be part of this programme. The UK medical community should receive further information of the ongoing discussion between AveXis and the UK regulatory authorities early in the new year. Eligible children in this age group are able to access nusinersen treatment.

Click here for more about the programme as announced so far.