Spinal Muscular Atrophy

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

Patient eligibility

The Pharmaceutical Benefits Scheme (PBS) subsidises treatment with nusinersen and risdiplam under the National Health Act 1953, section 100 for patients with SMA. Nusinersen is PBS subsidised for both symptomatic and pre-symptomatic SMA, whereas risdiplam is only PBS subsidised for symptomatic SMA.

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 and risdiplam.

Section 100 arrangements

Nusinersen and risdiplam

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

  • an approved private hospital
  • 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

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:

  • Lady Cilento 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.

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

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 treatment

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

Further information

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

Page last updated: 1 August 2021