Lysosomal Storage Disease Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)
全球的溶小體儲積症 (LSD)治療市場在2020年到2025年之間，將以8.2%的年複合成長率成長。 溶小體儲積症是因為遺傳基因缺陷所致，基因缺陷導致溶小體酵素缺乏或功能失常。而溶小體儲積症包括法布瑞氏症、高雪氏症、龐貝氏症、戴-薩克司氏症、天冬氨□葡萄糖胺尿症、貝敦氏症、胱胺酸症等。其中高雪氏症的特徵是神經系統併發症，是LSD疾病最普遍的症狀。其他症狀包括發燒、疼痛、痲痺、刺痛、手腳灼熱感、疲勞感、皮膚發紅或發紫、呼吸困難、暈眩等。
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.
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.
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.