Spinal Muscular Atrophy

The PBS subsidises nusinersen, onasemnogene abeparvovec, and risdiplam for patients with spinal muscular atrophy (SMA).

Patient eligibility

The Pharmaceutical Benefits Scheme (PBS) subsidises treatment with nusinersen, onasemnogene abeparvovec, and risdiplam under the National Health Act 1953, section 100 for patients with SMA.

Where the term ‘disease modifying therapy’ appears it refers to nusinersen and risdiplam. Where the term ‘gene therapy’ appears it refers to onasemnogene abeparvovec.

Nusinersen is PBS subsidised for both symptomatic and pre-symptomatic SMA, whereas risdiplam is only PBS subsidised for symptomatic SMA.

Onasemnogene abeparvovec is 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.

Patients must be eligible for the PBS and meet the relevant restriction criteria.

The Schedule of Pharmaceutical Benefits on the PBS website outlines restrictions for prescribing nusinersen, onasemnogene abeparvovec, and risdiplam.

Section 100 arrangements

Nusinersen, onasemnogene abeparvovec, and risdiplam

These items are only PBS subsidised for day admitted patients, non-admitted patients, or patients on discharge who are attending either:

  • an approved private hospital (nusinersen and risdiplam only)
  • a public participating hospital
  • a public hospital.

These items aren’t PBS subsidised for hospital in-patients. You must include the hospital name and provider number on the authority application form.

Treatment specifics

Nusinersen

To be eligible for PBS subsidised treatment with nusinersen for initial treatment, patients must be treated by or in consultation with a specialist medical practitioner who is both:

  • experienced in diagnosing and managing SMA
  • associated with a neuromuscular clinic of a recognised hospital in the management of SMA.

For continuing treatment, patients must either:

  • be treated by or in consultation with a specialist medical practitioner who is experienced in diagnosing and managing SMA and associated with a neuromuscular clinic of a recognised hospital in the management of SMA
  • be initiated by a specialist medical practitioner who is experienced in diagnosing and managing SMA and associated with a neuromuscular clinic of a recognised hospital in the management of SMA.

Hospitals recognised for managing SMA are:

  • Queensland Children’s Hospital - Brisbane
  • Royal Children’s Hospital - Melbourne
  • Monash Children’s Hospital - Melbourne
  • John Hunter Hospital - Newcastle
  • Sydney Children’s Hospital - Randwick
  • Children’s Hospital at Westmead
  • Adelaide Women and Children’s Hospital
  • Perth Children’s Hospital.

Onasemnogene abeparvovec

To be eligible for PBS subsidised treatment with onasemnogene abeparvovec for patients either untreated with disease modifying therapies or switching from disease modifying therapies to gene therapy, they must be treated by or in consultation with a specialist medical practitioner who is both:

  • experienced in diagnosing and managing SMA
  • associated with a neuromuscular clinic of a recognised hospital in the management of SMA.

Hospitals recognised for managing SMA are:

  • Queensland Children’s Hospital - Brisbane
  • Royal Children’s Hospital - Melbourne
  • Monash Children’s Hospital - Melbourne
  • John Hunter Hospital - Newcastle
  • Sydney Children’s Hospital - Randwick
  • Children’s Hospital at Westmead
  • Adelaide Women and Children’s Hospital
  • Perth Children’s Hospital.

Patients must be treated in a treatment centre that is each of the following:

  • recognised in managing SMA
  • accredited in using this gene technology by the relevant authority
  • has sourced or will source this product from an accredited supplier.

Accredited treatment centres and suppliers are those organisations accredited by the Gene Technology Regulator under section 92 of the Gene Technology Act 2000. The list of accredited organisations is available on the Department of Health website. These may be updated without notice.

Risdiplam

To be eligible for PBS subsidised treatment with risdiplam for both initial and continuing treatment, patients must be treated by or in consultation with a specialist medical practitioner who is both:

  • experienced in diagnosing and managing SMA
  • associated with a neuromuscular clinic.

Authority applications

Disease modifying therapy

Applying for initial treatment

Apply for initial authority approval to prescribe PBS subsidised nusinersen or risdiplam to treat SMA in writing and either:

All written applications must include the completed:

Applying for initial grandfather treatment

For patients who received non PBS subsidised risdiplam treatment before 1 August 2021 for symptomatic SMA, apply for initial grandfather authority approval in writing and either:

All written applications must include the completed:

Applying for continuing or change of treatment

Applications to continue or change authority approval to prescribe PBS subsidised nusinersen or risdiplam to treat SMA can be made either:

Gene therapy

Apply for authority approval to prescribe PBS subsidised onasemnogene abeparvovec to treat SMA in writing and either:

All written applications must include the completed:

You must state the weight of the patient in kilograms and request the appropriate product pack presentation with respect to the mix of 5.5 mL and 8.3 mL vials.

More information

Call the PBS Complex Drugs Programs enquiry line for further information.

Page last updated: 1 May 2022