The global gaucher disease (GD) drugs market size is expected to reach USD 1.71 billion by 2025, according to a new report by Grand View Research, Inc., exhibiting a CAGR of 1.1% during the forecast period. Growing prevalence of the disease and increasing research and development activities in the field are likely to help the market in gaining momentum over the coming years. With high unmet needs, there is a strong need for development of novel treatments with improved efficacy in neuronopathic complications, convenient dosage, and reduced cost.
Gaucher disease is a genetic metabolic disorder, caused by mutations in GBA gene. People with Gaucher disease produce low glucocerebrosidase (GCase), an enzyme required to degrade glucocerebroside. Excess accumulation of lipids in spleen, liver, kidney, brain, lungs, and bone marrow impedes the normal functioning of the organs.
In spite of being the most prevalent lysosomal storage disorder, Gaucher disease is a rare condition with an average prevalence of 1 in 100,000 individuals. However, certain ethnic races such as Ashkenazi Jews are more susceptible to this disorder. Ashkenazi Jews have an average disease prevalence of 1 in 500 individuals.
Currently, type 1 is the most common form of Gaucher disease. This condition is nonneuronopathic in nature, as it does not affect the central nervous system (CNS). Type 1 GD commanded more than 75.0% of the market in 2017. It is estimated to retain its position through 2025. Type 2 and type 3 are not as common as type 1.
On the basis of treatment, enzyme replacement therapy (ERT) was the leading revenue contributor in the market in 2017. The growth of the segment can be attributed to strong sales of Cerezyme and Vpriv. However, there has been an increase in inclination towards substrate replacement therapy (SRT), spearheaded by Cerdelga and Zavesca. ERT is mostly indicated for type 1 and type 3 (to address issues unrelated to the CNS), as replacement enzymes cannot penetrate blood brain barrier. Additionally, there is no effective treatment for type 2.
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Further key findings from the report suggest:
- Type 2 is the rarest of the three forms of GD, with average mortality rate of less than two years
- Enzyme replacement therapy dominated the treatment landscape, headed by Sanofi Genzyme’s Cerezyme. However, GD treatment is witnessing a shift towards substrate replacement therapy, which is anticipated to register a CAGR of over 7.5% during the forecast period
- The U.S. spearheaded the major markets, in terms of sales as well as disease burden. The country will continue to dominate the market through 2025, on account of widening patient base and easy accessibility to treatments
- Some of prominent companies operating in the market are Sanofi Genzyme, Shire, Pfizer, and Johnson & Johnson
- Sanofi Genzyme led the competitive space and is projected to continue holding prominent position in the market over the coming years, due to significant commercial performances of Cerezyme and Cerdelga
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