CADTH Canadian Drug Expert Committee recommendation: Onasemnogene abeparvovec (Zolgensma -- Novartis Pharmaceuticals Canada Inc.) indication: spinal muscular atrophy
Recommendation: The CADTH Canadian Drug Expert Committee (CDEC) recommends that onasemnogene abeparvovec be reimbursed for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene, only if the following conditions a...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
Canadian Agency for Drugs and Technologies in Health
March 26, 2021, 2021
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| Edition: | Version: Final |
| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | Recommendation: The CADTH Canadian Drug Expert Committee (CDEC) recommends that onasemnogene abeparvovec be reimbursed for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene, only if the following conditions are met: Conditions for ReimbursementInitiation criteria: 1. Genetic documentation of 5q SMA with biallelic mutations in the SMN1 gene. 2. Patients who are: 2.1. symptomatic or pre-symptomatic with one to three copies of the SMN2 gene 2.2. 180 days of age or younger 2.3. not currently requiring permanent feeding or ventilatory support (either invasive or non-invasive). Prescribing conditions: 1. Patient must be under the care of a specialist with experience in the diagnosis and management of SMA. 2. Reimbursement is limited to one lifetime administration of onasemnogene abeparvovec. Pricing conditions: 1. A reduction in price |
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| Physical Description: | 1 PDF file (18 pages) |