Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Pre-symptomatic spinal muscular atrophy (SMA)

Continuing/maintenance treatment of pre-symptomatic spinal muscular atrophy (SMA) with 3 copies of the SMN2 gene

Patient must have previously received PBS-subsidised treatment with this drug for this condition; or

Patient must be eligible for continuing PBS-subsidised treatment with nusinersen for this condition; AND

The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition; AND

The treatment must be given concomitantly with best supportive care for this condition; AND

The treatment must be ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug; AND

Must be treated by a specialist medical practitioner experienced in the diagnosis/management of SMA; or

Must be treated by a medical practitioner who has been directed to prescribe this benefit by a specialist medical practitioner experienced in the diagnosis/management of SMA; AND

Patient must not be undergoing treatment through this 'Continuing treatment' listing where the most recent PBS authority approval for this PBS indication has been for gene therapy; AND

Patient must have been 18 years of age or younger at the time of initial treatment with this drug; AND

Patient must be 2 years of age or older, and weigh 20 kg or more.

Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day.

In a patient who wishes to switch from PBS-subsidised nusinersen to PBS-subsidised risdiplam for this condition a wash out period may be required.