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Treatment for Spinal Muscular Atrophy

Nusinersen
Initial Treatment

Nusinersen may be considered medically necessary if all the following conditions are met:

Diagnosis of spinal muscular atrophy confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene as stated below:

deletion of both copies of the SMN1 gene OR

compound heterozygous mutations of the SMN1 gene (defined below):

pathogenic variant(s) in both copies of the SMN1 gene

pathogenic variant in one copy and deletion of the second copy of the SMN1 gene.

If patient is symptomatic, documentation of a genetic test confirms 2, 3 or 4 copies of the SMN2 gene; OR If patient is asymptomatic, documentation of a genetic test confirms minimum of 2 but less than 4 copies of the SMN2 gene.

Patient is not on permanent ventilator dependence

Initial approval is for 1 year, limited to the U.S. Food and Drug Administration approved dosing of 12 mg (5 mL) administered intrathecally per treatment with 4 loading doses; the first 3 loading doses should be administered at 14-day intervals. The fourth loading dose should be administered 30 days after the third dose. A maintenance dose should be administered once every 4 months thereafter; AND

The patient is not concurrently enrolled in a clinical trial for any experimental therapy for spinal muscular atrophy.

Prescribed by a neurologist with expertise in treating spinal muscular atrophy

Continuation of Treatment

Incremental reauthorization for nusinersen for 1 year may be considered medically necessary if the following conditions 1 and 2 are met:

The patient was previously approved for nusinersen based on criteria cited above

Documented evidence to support clinically meaningful improvement in motor milestones during previous treatment period.

Nusinersen is considered investigational for all other indications.

Concurrent use of nusinersen with onasemnogene abeparvovec-xioi and/or risdiplam is considered investigational.

Use of nusinersen after administration of onasemnogene abeparvovec-xioi is considered investigational.

Onasemnogene Abeparvovec-Xioi

Onasemnogene abeparvovec-xioi may be considered medically necessary if all of the following conditions are met:

Diagnosis of spinal muscular atrophy confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene as stated below

deletion of both copies of the SMN1 gene OR

compound heterozygous mutations of the SMN1 gene (defined below):

pathogenic variant(s) in both copies of the SMN1 gene

pathogenic variant in one copy and deletion of the second copy of the SMN1 gene.

Documentation of onset of symptoms consistent with clinical diagnosis of type I spinal muscular atrophy less than 6 months of age.

Documentation of a genetic test confirms no more than 2 copies of the SMN2 gene.

Patient is less than 6 months of age at the time of infusion of onasemnogene abeparvovec-xioi.

Patient does not have advanced spinal muscular atrophy (e.g., complete paralysis of limbs, permanent ventilator dependence).

Baseline anti-adeno-associated virus serotype 9 (AAV9) antibody titers < 1:50. Prescribed by a neurologist with expertise in treating spinal muscular atrophy. Repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi is considered investigational. Onasemnogene abeparvovec-xioi is considered investigational for all other indications. Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam is considered investigational. Use of nusinersen and/or risdiplam after administration of onasemnogene abeparvovec-xioi is considered investigational. Risdiplam Initial Treatment Risdiplam may be considered medically necessary if all the following conditions are met: Diagnosis of spinal muscular atrophy confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene as stated below: deletion of both copies of the SMN1 gene OR identification of pathogenic variant(s) in both copies of the SMN1 gene. If patient is symptomatic, documentation of a genetic test confirms 2, 3 or 4 copies of the SMN2 gene; OR If patient is asymptomatic, documentation of a genetic test confirms minimum of 2 but less than 4 copies of the SMN2 gene. Individual age is between 2 months to 25 years Patient is not on permanent ventilator dependence Initial approval is for 1 year, limited to the U.S. Food and Drug Administration approved dosing The patient is not concurrently enrolled in a clinical trial for any experimental therapy for spinal muscular atrophy. Prescribed by a neurologist with expertise in treating spinal muscular atrophy Continuation of Treatment Incremental reauthorization for risdiplam for 1 year may be considered medically necessary if the following conditions 1 and 2 are met: The patient was previously approved for risdiplam based on criteria cited above Documented evidence to support clinically meaningful stabilization or improvement in motor milestones during previous treatment period. Risdiplam is considered investigational for all other indications. Concurrent use of risdiplam with onasemnogene abeparvovec-xioi and/or nusinersen is considered investigational. Use of risdiplam after administration of onasemnogene abeparvovec-xioi is considered investigational

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