Spinraza Decision

Pharmaceuticals Benefits Advisory Committee (PBAC) announced its decision today against giving Spinraza a positive recommendation for all SMA sufferers aged over 19.

Specifically, this means that PBAC has not recommended the listing of nusinersen (Spinraza) be extended to include treatment of SMA patients with symptoms prior to 19 years of age. Neither has PBAC backed the proposed removal of the age limit of 18 years for initiation of treatment.

PBAC noted its requirement for further information regarding:

  • the Australian SMA patient population, especially patient numbers and variations
  • the cost effectiveness ratio of Spinraza as a treatment option
  • availability of further clinical evidence
  • definition of the appropriate adult population to receive Spinraza as a treatment
  • how Spinraza performs in the context of rapid developments in SMA treatments and diagnosis.

Naturally this decision is devastating to ourselves and our SMA community, but we recognise that this is a step in the journey and by no means the end of the road. It’s
heartening that, in delivering its decision, PBAC acknowledged both the high clinical need for effective treatments for adult SMA sufferers and the value of Spinraza in delivering treatment.

Such positive acknowledgement by PBAC is pleasing recognition of all our efforts to date in building recognition, support and life-changing treatment options for Australia’s SMA community. We have already proactively built on this acknowledgement too. We have attended urgent meetings with the PBAC deputy chair, PBAC consumer representative and Biogen representatives. PBAC considers it important to gain a treatment solution for adult sufferers of SMA.


PBAC requested and held a stakeholder meeting ahead of its announcement to gain:

  • critical information about pathways forward
  • required data
  • better understanding to support the proposal for Spinraza to be given a positive recommendation.

PBAC is also continuing to work with adult clinicians regarding treatment guidelines for the adult population.

We consider it positive that PBAC has acknowledged the high clinical need for effective treatments for adult SMA sufferers and the valuable role that Spinraza may play in delivering this. We will certainly continue forging forward on these strengths and invite you all to join us for a video conference in the new year to learn more about our proposed steps forward.


In the meantime, as a matter of urgency, we ask that all those who suffer SMA and are not members of SMA Australia to please register at the SMA Patient Registry. This will allow us develop a clear picture of SMA in Australia.

Click here to go to the patient registry

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