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Spinal muscular atrophy (SMA) Program in Pharmaceutical Benefits Scheme (PBS) 012-18053100



This document outlines details of PBS-subsidised nusinersen, onasemnogene abeparvovec and risdiplam for patients with spinal muscular atrophy (SMA).

SMA and listing dates

SMA is a rare neuromuscular disorder characterised by loss of motor neurons and progressive muscle wasting, often leading to early death.

Listing dates:

  • nusinersen - public hospital patients - 1 June 2018
  • nusinersen - private hospital patients - 1 November 2018
  • risdiplam - 1 August 2021
  • onasemnogene abeparvovec - public hospital patients - 1 May 2022

See Written Authority Required Drugs for more details.

Treatment specifics

The terms:

  • 'disease modifying therapy' refers to nusinersen or risdiplam
  • 'gene therapy' refers to onasemnogene abeparvovec.

Nusinersen and risdiplam are PBS-subsidised for the following populations:

  • Pre-symptomatic patients or symptomatic Type I/II/IIIa paediatric patients
  • Symptomatic Type IIIB/IIIC paediatric patients, and
  • Adult initiators (that is, patients aged 19 years or over who did not claim PBS-subsidy for a disease modifying treatment during childhood)

Onasemnogene abeparvovec is a PBS-subsidised gene therapy for both symptomatic Type 1 SMA and pre-symptomatic SMA for patients no older than 9 months of age. Treatment with onasemnogene abeparvovec is once only in a lifetime.

Enquiries

As of 1 November 2023, PBS Online allows automated payments for PBS medicines with a dispensed price of up to $9,999,999.99.

This means pharmacies can submit most PBS claims and receive full PBS reimbursement in a single automated payment via PBS online:

  • nusinersen payments are automated as of 1 November 2023
  • onasemnogene abeparvovec (Zolgensma®) payments are not yet automated

See Table 6 on the Process page for how to handle enquiries.

The Resources page contains:

  • application forms
  • contact details
  • restriction and item codes
  • the PBS schedule
  • Services Australia website link
  • Zolgensma® checklist

Online Pharmaceutical Benefits Scheme (PBS) Authorities System (OPA System)

Process telephone Authority approval application

Processing and National Demand Allocation (PaNDA)

Processing Complex Authority Required Listings

Processing Written Authority requests

Written Authority Required Drugs