Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p343
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 343/476)
Character Range: 3053104–3061665

greater than or equal to 16 hours per day.
                                                                                                                                            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).
                                                                                                                                            Signs and symptoms of spinal muscular atrophy in the context of this PBS restriction are:
                                                                                                                                            (i) Failure to meet or regression in ability to perform age‑appropriate motor milestones,
                                                                                                                                            (ii) Proximal weakness,
                                                                                                                                            (iii) Hypotonia,
                                                                                                                                            (iv) Absence of deep tendon reflexes,
                                                                                                                                            (v) Failure to gain weight appropriate for age,
                                                                                                                                            (vi) Any active denervation or chronic neurogenic changes found on electromyography,
                                                                                                                                            (vii) A compound muscle action potential below normative values for an age‑matched child.
                                                                                                                                            In this authority application, confirm:
                                                                                                                                            (1) the patient's medical history is consistent with a diagnosis of childhood onset spinal muscular atrophy,
                                                                                                                                            (2) which of the above (i to vii) (at least 1) were present during childhood,
                                                                                                                                            (3) the age of the patient (rounded to the nearest year) when the first sign/symptom was observed.
                                                                       C14372                                                               Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Compliance with Written Authority Required procedures
                                                                                                                                            Initial treatment
                                                                                                                                            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
                                                                                                                                            Patient must have experienced at least two of the defined signs and symptoms of SMA type I, II or IIIa prior to 3 years of age; AND
                                                                                                                                            The treatment must be given concomitantly with best supportive care 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 ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug.
                                                                                                                                            Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic.
                                                                                                                                            Patient must be untreated with gene therapy.
                                                                                                                                            Patient must be 18 years of age or under.
                                                                                                                                            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)