Fabry Disease Treatment MarketRegional Analysis
Europe Fabrydisease treatment market is expected to witness a drastic growth over theforecast period, owing to penetration of key players in key regions of Europewith their novel products for Fabry disease. For instance, Amicus TherapeuticsInc. launched Galafold (Migalastat) for treatment of Fabry disease in Italy (inMarch 2017) and in Spain (in January 2018)
In November 2018, JCRPharmaceuticals Co., Ltd, a Japan-based company launched biosimilar foragalsidase beta for treatment of Fabry disease. Launch of biosimilars isexpected to increase affordability of these therapies in key regions of AsiaPacific as biosimilars are quite cheaper than biologics, hence it is expectedto boost Asia Pacific Fabry disease treatment market growth.
Fabry disease also called asAnderson-Fabry disease and alpha-galactosidase-A deficiency is an X-linked,hereditary, lysosomal storage disease caused due to paucity of an enzyme αgalactosidase A (an enzyme required to metabolize lipids, fat-like substancesthat include oils, waxes, and fatty acids). The mutated gene leads theformation of lipids to harmful levels in various part of body such as theautonomic nervous system, cardiovascular system, eyes, and kidneys. Some of thecommon symptoms of this multisystem disorder include chronic pain,acroparesthesia, gastrointestinal disturbances, characteristic skin lesions(angiokeratomata), progressive renal impairment, cardiomyopathy, and stroke.
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Fabry Disease Treatment MarketDrivers
In recent past, variousregulatory bodies of key regions have approved novel medications for treatmentof Fabry disease. Approval and launch of such novel medications in the regionis expected to significantly support global Fabry disease treatment market growthover the forecast period.
For instance, in August 2018,Amicus Therapeutics, Inc., received the U.S. Food and Drug Administration (FDA)approval for its drug Galafold (migalastat). It is the first oral medicationindicated for the treatment of adults with Fabry disease.
The presence of robust medicationpipeline indicated for treatment of Fabry disease is also expected to be amajor factor fueling growth of global Fabry disease treatment market over theforecast period. Table mentioned below represents list of some novel therapiesin pipeline for Fabry disease treatment.
Furthermore, key players in themarket are majorly investing in development of novel therapies for Fabrydisease treatment, such strategic funding’s by various players is expected todrastically fuel global Fabry Disease treatment market growth over the forecastperiod. For instance, in February 2018, AvroBio Inc., closed US$ 60 millionfinancing to support the phase 2 study of its novel gene therapy, AVR-RD-01underdevelopment for the treatment of Fabry disease.
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Fabry Disease Treatment MarketRestraint
Fabry disease is a very raredisease affecting around 1 individual in 50,000 (according to the NationalFabry Disease Foundation), the diagnosis of Fabry disease is confirmed bydemonstrating an enzyme deficiency in males and by identifying the specific GLAgene mutation in males and females.
Hence, low diagnostic rate due tounavailability of such diagnostic procedures and trained professionals inemerging economies such as Africa, India, and China are expected to be majorfactor restraining global Fabry disease treatment market growth.
Fabry Disease Treatment MarketKey Players
Key players operating in globalFabry disease treatment market include: Amicus Therapeutics Inc., SanofiGenzyme (Sanofi S.A. company), Idorsia Pharmaceuticals Ltd., ProtalixBioTherapeutics, Inc., AvroBio Inc., JCR Pharmaceuticals Co., Ltd., ResverlogixCorp, and Ozmosis Research Inc.
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