Spinal Muscular Atrophy (SMA) Treatment Market Size, Share, Growth, Trends, and Global Industry Analysis: By Treatment (Gene Therapy), By Route of Administration (Oral, Intrathecal), By Disease Type (Type 1, Type 2, Type 3, Type 4) and Region Forecast 2020-2031

Spinal Muscular Atrophy (SMA) Treatment Market size was valued at US$ 3.4 billion in 2024 and is expected to reach US$ 8.7 billion by 2031, growing at a significant CAGR of 14.3% from 2025-2031. Due to increase in awareness regarding the disease (SMA), increase in the research for the rare disease treatment, initiative by government and private organizations to create awareness about the disease SMA, increase in number of initiatives for the treatment procedures of rare diseases, innovation of drugs and other therapies for the treatment and complex nature of the disease are the major factors that are driving the growth of the global spinal muscular atrophy (SMA) treatment market over the forecast timeframe.

Spinal muscular atrophy management is a category of hereditary illnesses that causes muscle degradation and compromises muscle function. It usually causes weakness and might even result in death. Motor neurons in the brain and spinal cord are affected by SMA. These motor neurons transmit electric and chemical impulses to and from voluntary muscles in the body, allowing for a variety of physical tasks such as walking, crawling, swallowing, and so on. Spinal muscular atrophy (SMA) management market is at the initial stage. Only one drug was approved for the management (as of 2016) and many of the drugs are still in clinical development. Market players are actively focusing on the drug development to enter into the market. For instance, big players such as Novartis AG is developing drug LMI070, which is in phase 2 clinical trial for management of spinal muscular atrophy type-1. Furthermore, companies are also focusing on acquisitions and mergers for expansion of their developmentl product portfolio.

Key Developments

• In January 2015, F. Hoffmann-La Roche AG acquired Trophos, a company focusing on development of novel drugs for orphan and neurodegenerative diseases. By this acquisition, Roche seeks to expand its development portfolio with olesoxime, spinal muscular atrophy drug being developed in phase-II clinical trials.
• In May 2017, Cytokinetics, Inc. pipeline spinal muscular atrophy drug candidate, CK-2127107 got orphan drug designation by Office of Orphan Products Development of U.S. FDA.
• In December 2016, Biogen Inc. received U.S. FDA approval for SPINRAZA (nusinersen) and European Commission (EC) granted market authorisation for the same in June 2017.