The sphingolipidoses are a group of inherited metabolic disorder caused due to deficiency of specific enzymes in the body. It is also known as lysosomal storage disorder. Lysosome is present inside the cell and is responsible for breakdown of cell substances that speeded up the reaction in the body. Lysosomal disorders occur due to absence of lysosomal enzymes in the body which results in development of toxic substance and thereby contributing in damage of organs. The disorder primarily hit the sites of sphingolipid catabolism like lysosomes of phagocytes, histiocytes or macrophages in the bone marrow, spleen and liver get affected. Sphingolipidoses is usually more prevalent amongst pediatric population. The degenerative pattern of disease is characterized by involvement of diffuse and progressive grey matter with psychomotor retardation. A cherry-red at the macula and optic atrophy are the common sign during the diagnosis of this disease. The disease is autosomal recessive in nature that results in enlarge liver and spleen and mental retardation. Specific lysosomal enzyme defects includes Niemann-Pick disease, Krabbe’s disease, Gaucher’s disease, Glycogen Storage disease II, Tay-Sachs disease and some other. All are rare metabolic disorders. Symptoms of sphingolipidoses includes fever, tingling, numbness, pain or burning in the feet and hand, tiredness, skin sores, dizziness and so on.
The sphingolipidoses treatment market is expected to witness a rapid growth during the forecast period due to factor such as allocation of twenty-three orphan drugs approved by FDA used for sphingolipidoses treatment. Moreover, rising incidence of lysosomal disease, increase in diagnostic rate, awareness about the rare disorders will create a demand to go for therapeutic treatment which will ultimately impact the market positively during the forecast period. Additionally, increase focus on R&D for diagnosis and drug development for sphingolipidoses treatment and implementation of orphan drugs that provide marketing and profitable benefit to pharmaceutical companies will also be contributing to drive the growth of sphingolipidoses treatment market. However, high cost of the available treatment, heterogeneity of the disease leading to misdiagnosis of sphingolipidoses and lack of diverse treatment option will hamper the market growth for sphingolipidoses treatment.
The global sphingolipidoses treatment market is segmented on the basis of indication, type of therapy, end user and region.
Based on indication, the sphingolipidoses treatment market is segmented as:
Based on therapy type, the sphingolipidoses treatment market is segmented as:
Based on end user, the sphingolipidoses treatment market is segmented as:
The sphingolipidoses treatment market is expected to have a significant growth in the market during the forecast period owing to increase incidence of lysosomal diseases and implementation of FDA approved orphan drugs in the market. According to the data published on Journal of Clinical and Experimental Hematology, the incidence of sphingolipidoses in non-Jewish population is 1:40,000 with type I Gauche accounts for 99% of cases. Based on the severity of disease, the enzyme replacement therapy is proven to be more effective as it helps in reducing the toxic material and organomegaly. Based on end user, the hospital segment is expected to account for major revenue during the forecast period as more number of therapy happens in the hospital.
In terms of region, the global sphingolipidoses treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to be dominant at the global level for sphingolipidoses treatment market due growing prevalence of disease in US and Canada. After North America, Europe is expected to hold the next major revenue share for sphingolipidoses treatment market owing to increase investment for R&D and clinical trials in European Union. Latin America will hold the significant share for sphingolipidoses treatment market due to increase government initiative and reimbursement policies in countries like Mexico and Brazil. Asia pacific is the emerging market and will hold the prominent share due to growing concern related to various diseases and various insurance payback policies. Middle East and Africa is expected to be the least lucrative region for sphingolipidoses treatment market due to less per capita expenditure on health and poor health healthcare accessibility.
Some of the key players for sphingolipidoses treatment market operating at the global level include Shire, Sanofi, Pfizer, Inc., Actelion Pharmaceuticals Ltd., BioMarin, Merck & Co., Inc., Raptor Pharmaceutical Corp., BioMarin Pharmaceutical Inc., Protalix Biotherapeutics Inc., Amicus Therapeutics, Inc., Novartis AG, Teva Pharmaceutical Industries Ltd. and others
The report on Sphingolipidoses Treatment market covers exhaustive analysis on:
Regional analysis for Sphingolipidoses Treatment market includes
Report on Sphingolipidoses Treatment market highlights: