The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Breaking news: CHMP recommends marketing authorisation of nusinersen for the treatment of SMA

    CHMP recommends marketing authorisation of nusinersen for the treatment of SMASMA Europe is delighted to announce that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the marketing approval of nusinersen, or Spinraza™ as it will now be called, for the treatment of patients with 5q spinal muscular atrophy (SMA).

    The CHMP opinion forms the basis for a European Commission decision on whether to grant a licence. The European Commission generally delivers its decision within three months.

    Marie-Christine Ouillade, SMA Europe President, says:

    “This recommendation by the CHMP is the first step towards a treatment for SMA. In order for this to be successful, each national member organisation will advocate its local authorities to accelerate availability to patients and reimbursement of nusineren in all European countries and support a broad label for all people with SMA”.

    At national level, the availability of the drug will depend on different issues such as its label, national reimbursement, health insurance systems and treatment readiness of centres.

    This treatment is the first in what is hoped will be a long list of treatments that will help to slow down and hopefully stop progression of the disease. Although there is now a treatment that gives hope we also know there is still a lot we don’t know about the condition and that we have not yet reached our final goal, a cure for all with spinal muscular atrophy, throughout their lifespan. SMA Europe will do its upmost to reach this goal.

    Nusinersen, developed by Ionis Pharmaceuticals Inc. and Biogen Inc., is the first drug to treat the underlying genetic cause spinal muscular atrophy. It is an antisense oligonucleotide designed to work on the SMN2 gene to increase the amount of functional SMN protein produced.

    More information

    Biogen’s community statementBreaking news - tiny
    CHMP press release

    If you have any questions, please contact your national SMA organisation as listed below:

    France: Association Francaise contre les Myopathies (AFM)
    Germany: Deutsche Gesellschaft für Muskelkranke (DGM)
    Iceland: FSMA á Íslandi
    Italy: Famiglie SMA
    Poland: Fundacja SMA
    Spain: FundAME
    Sweden: Nätverket för spinal muskelatrofi (NSMA)
    Switzerland: SMA Schweiz
    The Netherlands: Vereniging Spierziekten Nederland (VSN) or Prinses Beatrix Spierfonds
    UK: SMA Support UK & The SMA Trust
    Ukraine: Children with SMA (CSMA)