The Australian Government will list nusinersen on the Pharmaceutical Benefits Scheme (PBS) from 1 June 2018 for the treatment of Type 1, Type 2 and Type 3a spinal muscular atrophy (SMA)

Page last updated: 16 May 2018

Nusinersen (trade name Spinraza™) is marketed by the pharmaceutical sponsor Biogen Australia Pty Ltd (Biogen) as a treatment for SMA, a rare genetic neuromuscular disorder.

Biogen submitted a request to subsidise nusinersen through the Australian Government’s PBS for the treatment of Type 1, Type 2, and Type 3a SMA. It is expected that around 160 patients will receive treatment every year as a result of this listing.

The request was considered by the Pharmaceutical Benefits Advisory Committee (PBAC) in March 2018. The PBAC, an independent, expert group, makes recommendations to the Australian Government about PBS listings.

The PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of nusinersen for the treatment of paediatric patients with infantile-onset or childhood onset SMA with onset of symptoms prior to three years of age.

Patients would otherwise pay $367,850 a year for this medicine. The PBS listing will reduce the cost for patients to a maximum of $39.50 per script for general patient, and $6.40 for concessional patients.

The PBAC was satisfied that based on the clinical evidence available, nusinersen provides a significant improvement in efficacy over standard of care for the proposed patient population.

Biogen has agreed to open a compassionate access program for Nusinersen from 1 May 2018 ahead of the 1 June listing.

The PBAC’s decision can be found on the PBS website.