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Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA).
Read the full article on WikipediaSpinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Continuing/maintenance treatment with this drug of either symptomatic Type I, II or IIIa SMA, or, pre-symptomatic SMA (1 or 2 copies of the SMN2 gene) 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 ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug, AND The treatment must be given concomitantly with best supportive care for this condition. Treatment criteria: 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, 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. 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 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.
Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: 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. Treatment criteria: 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 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. Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day. 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). 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.
Symptomatic type IIIB/IIIC spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Continuing/maintenance treatment in a child or adult, but where treatment was initiated during childhood Treatment criteria: Patient must be undergoing continuation of existing PBS-subsidised treatment with this drug; OR Patient must be undergoing a change in prescribed SMA drug to this drug - the drug treatment being replaced was a PBS benefit initiated prior to the patient's 19th birthday for SMA type IIIB/IIIC, AND 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, AND Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS-subsidised disease modifying treatment. 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. Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day. 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.
Pre-symptomatic spinal muscular atrophy (SMA) Clinical criteria: Treatment Phase: Initial treatment with this drug of pre-symptomatic spinal muscular atrophy (SMA) Treatment criteria: 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, AND The condition must have genetic confirmation that there are 1 to 2 copies of the survival motor neuron 2 (SMN2) gene, AND The condition must be pre-symptomatic, AND The treatment must be given concomitantly with best supportive care for this condition, 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) 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.
“Risdiplam addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein. The protein is encoded by the SMN1 and SMN2 genes. SMA is caused by mutations in SMN1 that code for inactive forms of the protein. The activity of the SMN2 gene, which produces much smaller quantities of SMN, tends to determine the severity of disease.”
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