Spinal Muscular Atrophy (SMA) is a rare genetic neurodegenerative disease that primarily affects children. It is characterized by loss of spinal motor neurons, which are specialized cells that control muscle movement. The Spinal Muscular Atrophy (SMA) market across the five growth markets (5GM) Australia, China, India, Japan and South Korea in the Asia-Pacific (APAC) region is anticipated to grow at a compound annual growth rate of 23.3% from US$308.6m in 2018 to US$2.5bn in 2028. Increased uptake of the first disease-modifying Spinal Muscular Atrophy (SMA) therapies will drive the robust growth during the outlook period.

Biogen’s antisense therapy Spinraza (nusinersen) was first approved for the treatment of Spinal Muscular Atrophy (SMA) in Japan in June 2017 and has since been approved in Australia, South Korea and China. The approval of this drug is revolutionizing the management of the disease, with clinically meaningful improvements in motor function, achievement of milestones and survival observed for the first time in children with Spinal Muscular Atrophy (SMA).

Spinraza accounted for the vast majority of sales across the (5GM) Australia, China, India, Japan and South Korea in 2018 and sales are projected to remain strong throughout the early-to-mid forecast period (2018-2023). However, Spinraza is projected to lose its monopoly in the mid-to-late forecast period (2023-2028), as pipeline products launching from 2020 onwards are expected to capture significant market share.

Key opinion leaders express excitement regarding the potential of the late-stage pipeline to expand access to therapy and address key unmet needs. In particular, Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec-xioi) represents a potentially transformative treatment option, as it is administered by a one-time intravenous infusion. This represents a strong differentiating factor from Spinraza, which requires intrathecal administration multiple times per year.

Zolgensma, which has already received approval in the US, is expected to emerge as the market leader in the APAC region by 2028 with sales of US$1.3bn across the (5GM) Australia, China, India, Japan and South Korea. Additionally, the launch of Roche’s risdiplam, an SMN2 splicing modifier, is also eagerly anticipated owing to the drug’s convenient oral administration route.

Although Spinraza’s approval represented a major breakthrough in the field, there is still an unmet need for additional therapies that employ novel mechanisms of action and administration routes, which can translate into improvements in efficacy, accessibility, ease of use, and patient compliance.

The latest research report “Spinal Muscular Atrophy: Forecast in Asia-Pacific Markets to 2028” helps in answering the following question with regards to Spinal Muscular Atrophy (SMA) and its therapeutic market in Asia Pacific.

  • How Spinal Muscular Atrophy (SMA) is currently managed and does this vary by subtype?
  • To what extent will the launch of pipeline products over the forecast period fulfill the unmet needs in the market?
  • How will the launch of pipeline products impact Spinraza?
  • How will growth vary by market?


  • Overview of SMA, including epidemiology, etiology, pathophysiology, classification, diagnosis, and disease management.
  • Market Data including annualized SMA therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in fours patient segments (Type 1, 2, 3, and 4) forecast from 2018 to 2028.
  • Key topics covered include unmet needs and opportunity assessment, R&D strategies and clinical trial design.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for SMA therapy. The most promising candidates in Phase II and III development are profiled.
  • Analysis of the current and future market competition in the SMA therapeutics market: Insightful review of the industry drivers and barriers and in-depth analysis of trends within each of the 5GM.

Reasons To Buy

The report will enable you to -

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies most active within the SMA APAC landscape.
  • Develop business strategies by understanding the trends shaping and driving the SMA APAC market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the SMA APAC market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.