Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p346
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 346/476)
Character Range: 3076926–3085092

be undergoing initial PBS‑subsidised treatment with this drug for untreated disease; OR
                                                                                                                                            Patient must be undergoing initial PBS‑subsidised treatment, but the patient has initiated treatment via non‑PBS supply (e.g. clinical trial, sponsor compassionate access); AND
                                                                                                                                            Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS‑subsidised disease modifying 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 not be receiving invasive permanent assisted ventilation in the absence of a potentially reversible cause while being treated with this drug.
                                                                                                                                            Invasive permanent assisted ventilation means ventilation via tracheostomy tube for 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) Any active denervation or chronic neurogenic changes found on electromyography,
                                                                                                                                            (vi) 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 type IIIB/IIIC spinal muscular atrophy,
                                                                                                                                            (2) which of the above (i to vi) (at least 1) were present after their 3rd birthday, but before their 19th birthday,
                                                                                                                                            (3) the age of the patient (rounded to the nearest year) when the first sign/symptom was observed.
                                                                                                                                            The quantity of drug and number of repeat prescriptions prescribed is to be in accordance with the relevant 'Note' attached to this listing.
                                                                                                                                            The approved Product Information recommended dosing is as follows:
                                                                                                                                            (i) 16 days to less than 2 months of age: 0.15 mg/kg
                                                                                                                                            (ii) 2 months to less than 2 years of age: 0.20 mg/kg
                                                                                                                                            (iii) 2 years of age and older weighing less than 20 kg: 0.25 mg/kg
                                                                                                                                            (iv) 2 years of age and older weighing 20 kg or more: 5 mg
                                                                                                                                            In this authority application, state which of (i) to (iv) above applies to the patient. Based on (i) to (iv), prescribe up to:
                                                                                                                                            1 unit where (i) applies;
                                                                                                                                            2 units where (ii) applies;
                                                                                                                                            3 units where (iii) applies;
                                                                                                                                            3 units where (iv) applies.
                                                                       C14420                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Authority