Lysosomal Storage Disease Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

The lysosomal storage disease treatment market is expected to register a CAGR of 8.2% during the forecast period. Lysosomal storage diseases are a group of rare inherited metabolic disorders that result from the lack or malfunctioning of lysosomal enzymes. Some of the lysosomal storage diseases are Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, aspartylglucosaminuria, Batten disease, cystinosis, and others. Among them Gaucher disease is the most common type of lysosomal disorder which is characterized by neurological complication. Some of the symptoms such as fever, pain, numbness, tingling, or burning in the hands and feet, tiredness, red or purple skin sores, trouble in breathing, dizziness and so on.

Currently, there are twenty-three orphan drugs approved for the treatment of 11 lysosomal storage diseases. Among them four diseases have multiple therapeutics approved, i.e. Fabry disease, cystinosis, Gaucher disease, and Pompe disease. Neuronal ceroid lipofuscinosis 2 (CLN2), Lysosomal acid lipase deficiency (LAL-D), and Mucopolysaccharidosis type I, II, IVA, VI, VII have one compound approved for treatment by the FDA.

According to the National Gaucher Foundation, Gaucher disease occurs in 1 in 50,000 to 100,000 people in the general population. Type 1 Gaucher disease is the most common form of the disorder. Gaucher disease can affect anyone, occurring in up to 1 in 40,000 live births in the general population.

Increase in incidence of lysosomal diseases, growing awareness among people regarding rare lysosomal storage disease and increasing research and development for diagnosis and drug development for treatment of lysosomal diseases are the key driving factors in lysosomal storage disease treatment market.

Key Market Trends

Enzyme Replacement Therapy Segment is Expected to Hold a Major Market Share in the Lysosomal Storage Disease Treatment Market

• Enzyme replacement therapy is a treatment which replaces an enzyme that is absent or deficient in the body. Some of the available enzyme replacement therapy for lysosomal storage diseases are Velaglucerase Alfa, Taliglucerase Alfa, Agalsidase beta, Laronidase, Imiglucerase and among others.
• National Institute of Neurological Disorders and Stroke (NINDS), estimates that 5,000 to 10,000 people have pompe disease worldwide. In addition, incidence of infantile onset pompe disease varies between ethnic groups and regions. In some countries, such as in African Americans, the incidence is as high as one in 14,000 people.
• Enzyme replacement therapy segment holds a significant market share in the lysosomal storage disease treatment market and is anticipated to show similar trend over the forecast period due to improving organ function and effective in reducing toxic storage material in patients suffering from lysosomal disorders.
• Increase in incidence of lysosomal diseases and increasing research and development for diagnosis and drug development for treatment of lysosomal diseases are the key driving factors in the enzyme replacement therapy segment.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

North America is expected to hold a major market share in the global lysosomal storage disease treatment market due to increasing incidence of lysosomal storage disorders in this region. According to the National Organization for Rare Disorders (NORD), approximately 6,000 individuals are affected with Gaucher disease in the United States. Moreover, Type 1 Gaucher disease is the most common form of the disease in western countries, making up roughly 95 percent of patients. Furthermore, increasing research and development for diagnosis and drug development for treatment of lysosomal diseases and presence of well-established healthcare infrastructure is also fueling the growth of the overall regional market to a large extent.

Competitive Landscape

The lysosomal storage disease treatment market is consolidated competitive and consists of a few major players. In terms of market share, few of the major players are currently dominating the market. Some of the leading market players include Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Therapeutics, Inc, Alexion Pharmaceuticals, Inc, Sigilon Therapeutics, Inc and Orphazyme A/S.

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