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Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Initial treatment - Loading doses Treatment criteria: 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 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 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 not exceed four loading doses (at days 0, 14, 28 and 63) under this restriction, AND Patient must not have received PBS-subsidised gene therapy. Population criteria: 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) A compound muscle action potential below normative values for an age-matched child. Defined signs and symptoms of type II SMA are: i) Onset between 6 and 18 months; and ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or iii) Proximal weakness; or iv) Weakness in trunk righting/derotation; or v) Hypotonia; or vi) Absence of deep tendon reflexes; or vii) Failure to gain weight appropriate for age; or viii) Any active chronic neurogenic changes; or ix) A compound muscle action potential below normative values for an age-matched child. Defined signs and symptoms of type IIIa SMA are: i) Onset between 18 months and 3 years 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. Application for authorisation of initial treatment must be in writing and must include: (a) details of the proposed prescription; and (b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following: (i) specification of SMA type (I, II or IIIa); and (ii) sign(s) and symptom(s) that the patient has experienced; and (iii) patient's age at the onset of sign(s) and symptom(s).
“Nusinersen (contained in SPINRAZA) increases the proportion of exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts by binding to an intronic splice silencing site (ISSN1) found in intron 7 of the SMN2 pre messenger ribonucleic acid (pre-mRNA).”
“135–177 days (in CSF), 63–87 days (in plasma)”
Working under the parallel aged-care framework? Aged-care equivalent →
Spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Continuing/maintenance treatment of either symptomatic Type I, II or IIIa SMA, or of a patient commenced on this drug under the pre-symptomatic SMA (1 or 2 copies of the SMN2 gene) listing Treatment criteria: 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; or initiated 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, 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 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. Population criteria: 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.
Pre-symptomatic spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Initial treatment of pre-symptomatic spinal muscular atrophy (SMA) with 1 or 2 copies of the SMN2 gene - Loading doses Treatment criteria: 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 1 to 2 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 not have received PBS-subsidised gene therapy. Population criteria: 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) details of the proposed prescription; 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)
Symptomatic type IIIB/IIIC spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Initial PBS-subsidised treatment in a child 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. Population criteria: Patient must be of an age that is prior to their 19th birthday at the time of this authority application, AND Patient must have SMA type III where the onset of signs/symptoms of SMA first occurred after their 3rd birthday, but before their 19th birthday (SMA type IIIB/IIIC). Treatment criteria: 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, AND Patient must be undergoing initial PBS-subsidised treatment for untreated disease - prescribe up to 3 repeat prescriptions to enable dosing occurring at days: 0 (original prescription), 14 (repeat 1), 28 (repeat 2), 63 (repeat 3) (i.e. the loading doses); 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) - prescribe zero repeat prescriptions where loading doses are complete, AND Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS-subsidised disease modifying treatment. The authority application must be made in writing and must include: (1) details of the proposed prescription; 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.
Curated subset. The full adverse-effect list is in the TGA Product Information; click any citation above to open it.