Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p282
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 282/476)
Character Range: 2618905–2626897

for this PBS indication has been for gene therapy.
                                                                                                                                            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 risdiplam for this condition; AND
                                                                                                                                            The treatment must not be in combination with PBS-subsidised treatment with risdiplam 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.
                                                                                                                                            Patient must have been 18 years of age or younger at the time of initial treatment with this drug.
                                                                                                                                            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 risdiplam to PBS-subsidised nusinersen for this condition a wash out period may be required.
                                                                       C15116                                                               Pre-symptomatic spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Initial treatment of pre-symptomatic spinal muscular atrophy (SMA) with 3 copies of the SMN2 gene - Loading doses
                                                                                                                                            Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
                                                                                                                                            The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
                                                                                                                                            The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
                                                                                                                                            The condition must be pre-symptomatic SMA, with genetic confirmation that there are 3 copies of the survival motor neuron 2 (SMN2) gene; AND
                                                                                                                                            The treatment must be given concomitantly with best supportive care for this condition; AND
                                                                                                                                            The treatment must not exceed four loading doses (at days 0, 14, 28 and 63) under this restriction; AND
                                                                                                                                            Patient must be untreated with gene therapy.
                                                                                                                                            Patient must be aged under 36 months prior to commencing treatment.
                                                                                                                                            Application for authorisation of initial treatment must be in writing (lodged via postal service or electronic upload) and must include:
                                                                                                                                            (a) a completed authority prescription form; and
                                                                                                                                            (b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
                                                                                                                                            (i) confirmation of genetic diagnosis of SMA; and
                                                                                                                                            (ii) a copy of the results substantiating the number of SMN2 gene copies determined by quantitative polymerase chain reaction (qPCR) or multiple ligation dependent probe amplification (MLPA)
Ocrelizumab                                                            C7386                                                                Multiple sclerosis                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                    Compliance