LATAM Fabry Disease Market, by Therapy Type (Enzyme Replacement Therapy (Agalsidase Beta (Fabrazyme), Agalsidase Alfa (Replagal), Pegunigalsidase Alfa (PRX-102, Phase III)) Chaperon Therapy (Migalastat (Galafold Phase III)), Substrate Reduction Therapy (Lucerastat (Phase III), Venglustat (Phase III))), by Route of Administration (Oral, Parenteral), by Distribution Channel (Hospitals Pharmacies, Online pharmacies, Retail Pharmacies), and by Country (Brazil, Argentina, Mexico, Rest of Latin America) - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2030
Fabry disease is an inherited disorder which is caused by accumulation for globotriaosylceramide, a type of fat, in the body’s cells. Fabry disease symptoms affect body parts which include a decreased ability to sweat, events of pain in hands and feet, difficulties with the gastrointestinal system, etc. Diagnosis for fabry disease includes test such as Deoxyribonucleic acid sequence analysis, enzyme assay analysis, etc.
Treatment for fabry disease includes intravenously administered enzyme replacement therapy or oral chaperone therapy, along with this it may also need lifestyle modifications and prophylactic medications. Sometimes doctors also refer drugs to relieve pain, medicine for stomach problems, blood thinners or other drugs for an irregular heartbeat or other heart problems, blood pressure medicine, which also helps to protect kidneys. If fabry disease has caused serious kidney damage, dialysis or a kidney transplant is needed for treatment.
Market Dynamics
Survey regarding achievements or learning about fabry disease, is expected to drive growth of the LATAM fabry disease market. For instance, according to data published on June 20, 2022, by Orphanet Journal of Rare Diseases, a team of researchers had done 20 years of Fabry Outcome Survey that focused to broaden the understanding of fabry disease such as to know about the insights, achievements, and learning more about the development of fabry disease, effects of treatment, etc. according to same Route of Administration population suffering from fabry disease has reported cardiovascular problem and it was more common type of problem reported in male and female in with fabry disease in Latin America.
Updated guidelines by regulatory bodies for the development of drugs used in the treatment of fabry disease is expected to drive the LATAM fabry disease market growth. For instance, in August, 2019, the U.S. Food and Drug Administration announced the availability the draft guidance for the industries, to develop drugs for the treatment of fabry disease. This draft guidance describes the FDA’s current recommendations regarding eligibility criteria, trial design considerations, and efficacy endpoints to be used in clinical development programs of investigational drugs to treat fabry disease.
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