Document ID: chunk:federal_register_of_legislation:F2024L01395:schedule:1:p19
Version: federal_register_of_legislation:F2024L01395
Segment Type: schedule
Provision Reference: sch 1 (pt 19/20)
Character Range: 63214–66551

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.
              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.
              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 nusinersen to PBS-subsidised risdiplam for this condition a wash out period may be required.
              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.
   C15990     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
              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.
              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;