Spinal Muscular Atrophy is a group of neuromuscular disorders that lead to the loss of motor neurons along with progressive muscle degeneration. The condition is the result of a genetic defect in the SMN1 gene, which is necessary for SMN protein production – responsible for the survival of motor neurons. Due to the defect, correct amounts of the protein are not formed, leading to degrading communications between the brain and skeletal muscles.
The Spinal Muscular Atrophy (SMA) market is predicted to grow at an annual compound growth rate of 23.2% from US $308.6m in 2018 to US $2.5bn in 2028, across the five growth markets(5GM) – Australia, Japan, China, India and South Korea.
An antisense therapy, Spinraza, played a large role in the sales across the 5GM and is anticipated to stay strong throughout early-to-mid forecast period (2018-2023). The approval of the drug is revolutionizing the disease’s management, with betterment in the neuron function and higher survival rates observed in children with Spinal Muscle Atrophy.
Spinraza (nusinersen) is a relatively new medication approved by the Food and Drug Administration, in 2017, for the treatment of all forms of SMA. The drug was initially approved in Japan but is now available in Australia, South Korea and China as well.
The medication significantly slows the disease’s progression and also improves the overall strength of the patient. Multiple studies and clinical trials indicated that the drug was beneficial for about 40% of the patients who used it.
Spinraza is injected into the fluid surrounding the spinal cord through an epidural needle – inserted into the lower back. The “intrathecal injection” is performed during a lumbar puncture or spinal tap and takes around 15-30 minutes. The whole procedure of injecting and recovery, however, can go up to 2 hours.
Even though, the drug’s sale are predicted to stay strong up to early-to-middle range, it might lose its monopoly later on (2023 – 2028) due to the launch of pipeline products from 2020 that are expected to capture a significant market share.
Zolgensma, a gene therapy for SMA has already been approved in US and is expected to emerge as a market leader in the APAC region by 2028 with estimated sales of US $1.3bn across the 5GM. Furthermore, the launch of Roche’s risdiplam, an SMN2 splicing modifier, is also anticipated to provide a convenient oral administration route of the drug.
Zolgensma is a prescription gene therapy used to treat children less than 2 years old, suffering from SMA, and is given as a one-time infusion into the vein. Even though it’s effective, the drug can cause acute serious liver injury and nausea due to the elevated amounts of liver enzymes. Platelet count may also decrease following the infusion and hence should be kept in check.
A recent research report, “Spinal Muscular Atrophy: Forecast in Asia-Pacific Markets to 2028” gives more details about the scope of SMA market in the future. Following are a few topics, the report effectively covers:
- Management of SMA and its subtypes
- Extent to which the launch of pipeline products will cater to the needs of the market
- Impact of the launch of pipeline products on Spinraza
- Variation of growth by market