Fabry disease also called as Anderson-Fabry disease and alpha-galactosidase-A deficiency is an X-linked, hereditary, lysosomal storage disease caused due to paucity of an enzyme α galactosidase A (an enzyme required to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids). The mutated gene leads the formation of lipids to harmful levels in various part of body such as the autonomic nervous system, cardiovascular system, eyes, and kidneys. Some of the common symptoms of this multisystem disorder include chronic pain, acroparesthesia, gastrointestinal disturbances, characteristic skin lesions (angiokeratomata), progressive renal impairment, cardiomyopathy, and stroke.
Fabry Disease Treatment Market Drivers
In recent past, various regulatory bodies of key regions have approved novel medications for treatment of Fabry disease. Approval and launch of such novel medications in the region is expected to significantly support global Fabry disease treatment market growth over 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 medication indicated for the treatment of adults with Fabry disease.
The presence of robust medication pipeline indicated for treatment of Fabry disease is also expected to be a major factor fueling growth of global Fabry disease treatment market over the forecast period. Table mentioned below represents list of some novel therapies in pipeline for Fabry disease treatment.
Furthermore, key players in the market are majorly investing in development of novel therapies for Fabry disease treatment, such strategic funding’s by various players is expected to drastically fuel global Fabry Disease treatment market growth over the forecast period. For instance, in February 2018, AvroBio Inc., closed US$ 60 million financing to support the phase 2 study of its novel gene therapy, AVR-RD-01 underdevelopment for the treatment of Fabry disease.
Fabry Disease Treatment Market Regional Analysis
Europe Fabry disease treatment market is expected to witness a drastic growth over the forecast period, owing to penetration of key players in key regions of Europe with their novel products for Fabry disease. For instance, Amicus Therapeutics Inc. launched Galafold (Migalastat) for treatment of Fabry disease in Italy (in March 2017) and in Spain (in January 2018)
In November 2018, JCR Pharmaceuticals Co., Ltd, a Japan-based company launched biosimilar for agalsidase beta for treatment of Fabry disease. Launch of biosimilars is expected to increase affordability of these therapies in key regions of Asia Pacific as biosimilars are quite cheaper than biologics, hence it is expected to boost Asia Pacific Fabry disease treatment market growth.
Fabry Disease Treatment Market Restraint
Fabry disease is a very rare disease affecting around 1 individual in 50,000 (according to the National Fabry Disease Foundation), the diagnosis of Fabry disease is confirmed by demonstrating an enzyme deficiency in males and by identifying the specific GLA gene mutation in males and females.
Hence, low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals in emerging economies such as Africa, India, and China are expected to be major factor restraining global Fabry disease treatment market growth.
Fabry Disease Treatment Market Key Players
Key players operating in global Fabry disease treatment market include: Amicus Therapeutics Inc., Sanofi Genzyme (Sanofi S.A. company), Idorsia Pharmaceuticals Ltd., Protalix BioTherapeutics, Inc., AvroBio Inc., JCR Pharmaceuticals Co., Ltd., Resverlogix Corp, and Ozmosis Research Inc.
Fabry Disease Treatment Market Taxonomy:
The global Fabry disease treatment market is segmented on the basis of drugs, molecule type, route of administration, distribution channel, and region
- Approved Drugs
- Agalsidase Beta (Fabrazyme/Replagal)
- Migalastat (Galafold)
- Pipeline Drugs
By Molecule Type-
- Small Molecule
- Large Molecule (Biologics)
By Route of Administration-
By Distribution Channel-
- Hospitals Pharmacies
- Retail Pharmacies
- Online pharmacies
- North America
- Latin America
- Asia Pacific
- Middle East