GM1 Gangliosidosis is an inherited central nervous system disorder that destroys nerve cells in the spinal cord and brain. GM1 gangliosidosis is classified mainly into three types depending upon the signs and symptoms of the disorder. Type 1 GM1 gangliosidosis includes severe signs and symptoms such as mental regression and development of an exaggerated startle reaction to loud noises. Type 1 GM1 gangliosidosis usually occurs in newborns under 6 months of age. Type 2 GM1 gangliosidosis is also known as the late infantile and juvenile form. Children suffering from this usually exhibit symptoms such as mental regression till the age of 18. In type 3 GM1 gangliosidosis (also known as the adult or chronic form), the symptoms are usually seen in adults but exhibit a very low disease spectrum.
The global GM1 gangliosidosis treatment market size is estimated to be valued at US$ 142.1 million in 2025 and is expected to exhibit a CAGR of 35.6 % during the forecast period (2025-2030).
Figure 1. Global GM1 Gangliosidosis Treatment Market Value (US$ Mn), 2025 - 2030
GM1 gangliosidosis is a rare central nervous system disorder and can develop throughout the lifetime in diagnosed individuals. For instance, in 2019, according to World Health Organization WHO data, around 2,000 patients suffer from GM1 ganglioside across the world. According to the same data source, incidence GM1 gangliosidosis accounts for around 0.5-1.0/100,000 births across the globe.
|Base Year:||2019||Market Size in 2025:||US$ 142.1 Mn|
|Historical Data for:||2016 to 2019||Forecast Period:||2020 to 2027|
|Forecast Period 2025 to 2030 CAGR:||36.5%||2030 Value Projection:||US$ 651.7 Mn|
Axovant Gene Therapies Ltd, Passage Bio and Lysogene.
|Restraints & Challenges:||
Global GM1 Gangliosidosis Treatment Market – Impact of Coronavirus (COVID-19)
Following the outbreak of COVID-19 in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization has declared it a public health emergency. According to the World Health Organization’s report, the manifestation of the coronavirus (COVID-19) has resulted in more than 52.7 million infected individuals worldwide as of November 13, 2020.
COVID-19 has affected each and every market across the world and has also impacted the global GM1 gangliosidosis treatment market in the following ways; by directly affecting production and demand, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to the lockdown, several countries such as China, India, Saudi Arabia, UAE, Egypt, and others are facing problems with regards to the transportation of drugs from one place to another.
Furthermore, players operating in the global GM1 gangliosidosis market are facing major challenges on various fronts due to the COVID-19 pandemic. The major challenges include supply of raw materials for manufacturing drug formulations due to irregularities in transportation facility. Moreover, distributors of products are experiencing irregular demand for products from the retailers due to increasing patient population suffering from COVID-19 and other life threatening disorders.
Figure 2. Global GM1 Gangliosidosis Treatment Market Share (%), by Region, 2025
Among region, the North America GM1 gangliosidosis market is expected to witness significant growth during the forecast period, owing to high disease population in the U.S. For instance, in 2019, according to National Center for Biotechnology Information (NCBI) data, the GM1 gangliosidosis accounts for around 500 patients, which is expected to drive growth of the U.S market.
Major players operating in the global GM1 Gangliosidosis Treatment market are Axovant Gene Therapies Ltd. and Passage Bio and Lysogene.