Pharmacoeconomic review report (resubmission): Nusinersen (Spinraza) (Biogen Canada Inc.) indication : treatment of patients with 5q spinal muscular atrophy
Those with SMA type II achieve the milestone of sitting unsupported, but never walk independently; symptoms generally appear between six and 18 months after birth and most patients will survive past the age of 25, with life expectancy improved by aggressive supportive care. SMA type III makes up app...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
CADTH
April 2019, 2019
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| Edition: | Final (with redactions) |
| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | Those with SMA type II achieve the milestone of sitting unsupported, but never walk independently; symptoms generally appear between six and 18 months after birth and most patients will survive past the age of 25, with life expectancy improved by aggressive supportive care. SMA type III makes up approximately 10% to 20% of SMA cases3 and presents between 18 months of age and adulthood. These patients are able to walk independently at some point in their lives and typically have a normal life expectancy. SMA type IV constitutes a very small proportion of SMA cases, has an adult onset, and is the mildest form of the disease. Although muscle weakness is present, these patients retain the ability to walk, have a normal life expectancy, and do not suffer from respiratory or nutritional issues. Nusinersen (Spinraza) is a solution for intrathecal injection, indicated for the treatment of 5q SMA. It is available as a single-use solution in a 5 mL vial size (12 mg) administered intrathecal by lumbar puncture. The recommended dose is initial treatment with four loading doses, with the first three loading doses administered at 14-day intervals (day 0, day 14, and day 28), and a final loading dose approximately 30 days after the third loading dose (day 63); maintenance treatment is 12 mg every four months. At the marketed price of $118,000 per 5 mL vial, the annual cost of treatment with nusinersen ranges from $354,000 for maintenance treatment (three doses) to $708,000 in the first year (six doses). The manufacturer's listing request is per the Health Canada indication. As part of a resubmission the manufacturer provided new clinical information relating to different subpopulations with SMA (see CADTH Common Drug Review [CDR] Clinical Report). Spinal muscular atrophy (SMA) is a severe neuromuscular disease and is the leading genetic cause of infant death. It is characterized by the degeneration of alpha motor neurons in the anterior horn of the spinal cord, leading to progressive muscle weakness. The most common form of SMA, 5q SMA, makes up more than 95% of all cases and is an autosomal recessive disorder caused by homozygous deletion or deletion and mutation of the alleles of the survival motor neuron 1 gene. SMA is a rare disease and estimates of its incidence and prevalence vary between studies. The incidence of SMA is often cited as being approximately 10 in 100,000 live births. Four clinical subtypes of SMA are described: SMA type I makes up about 60% of SMA diagnoses where patients show symptoms before six months of age, never achieve the motor milestone of sitting unsupported, and generally do not survive past two years of age due to respiratory failure. The manufacturer, however, did not provide a revised economic submission nor did it provide any discussion relating to the new clinical information and how this may impact the findings of the economic review based on the original submission. Thus, the CDR economic review remains unchanged with no further data provided to refute the original issues or limitations identified |
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| Physical Description: | 1 PDF file (35 pages) illustrations |