Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p301
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 301/476)
Character Range: 2748823–2756929

accredited in the use of this gene technology by the relevant authority, (iii) will(has) source(d) this product from an accredited supplier, as specified in the administrative notes to this listing; AND
                                                                                                                                            Patient must be undergoing treatment with this pharmaceutical benefit once only in a lifetime; AND
                                                                                                                                            Patient must not be undergoing treatment with this pharmaceutical benefit through this listing where prior treatment has occurred with any of: (i) nusinersen, (ii) risdiplam.
                                                                                                                                            Patient must be no older than 9 months of age; AND
                                                                                                                                            Patient must have symptomatic Type 1 SMA; OR
                                                                                                                                            Patient must have pre‑symptomatic SMA.
                                                                                                                                            The authority application must be made in writing and must include:
                                                                                                                                            (1) a completed authority prescription form; and
                                                                                                                                            (2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).
                                                                                                                                            Prescribing Instructions:
                                                                                                                                            In the relevant PBS Authority Application form, specify the following:
                                                                                                                                            (i) the SMA type being treated: symptomatic Type 1 SMA, or, pre‑symptomatic SMA;
                                                                                                                                            (ii) for Type 1 SMA, the signs/symptoms that the patient has experienced, together with the patient's age at the onset of these signs/symptoms.
                                                                                                                                            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.
                                                                                                                                            Confirm that genetic testing has been completed to demonstrate the following in support of an SMA diagnosis:
                                                                                                                                            (i) 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; or
                                                                                                                                            (ii) deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variance in the remaining single copy of the SMN1 gene.
                                                                                                                                            If the condition is pre‑symptomatic SMA, confirm that there is genetic test finding that substantiates the number of SMN2 gene copies determined by quantitative polymerase chain reaction (qPCR) or multiple ligation dependent probe amplification (MLPA).
                                                                                                                                            Quote the date, pathology provider name and any unique identifying serial number/code that links the genetic test result to the patient.
                                                                                                                                            Defined signs and symptoms of type I SMA are:
                                                                                                                                            i) Onset before 6 months of age; and
                                                                                                                                            ii) Failure to meet or regression in ability to perform age‑appropriate motor milestones; or
                                                                                                                                            iii) Proximal weakness; or
                                                                                                                                            iv) Hypotonia; or
                                                                                                                                            v) Absence of deep tendon reflexes; or
                                                                                                                                            vi) Failure to gain weight appropriate for age; or
                                                                                                                                            vii) Any active chronic neurogenic changes; or
                                                                                                                                            viii) A compound muscle action potential below normative values for an age‑matched child.
                                                                       C15460                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Use occurring after treatment with at least one disease modifying therapy for this condition (i.e. switching from nusinersen/risdiplam to onasemnogene abeparvovec)
                                                                                                                                            The treatment must be given concomitantly with best supportive care for this condition; AND
                                                                                                                                            The treatment must not be