Agalsidase beta, sold under the brand name Fabrazyme by the company Sanofi Genzyme, is another recombinant form of alpha-galactosidase. Like replagal, it received approval in the EU in 2001. In 2003, it was the first treatment for Fabry disease to be approved by the FDA.Clinically, the two treatments are generally similar in effectiveness and safety, however they have never been compared directly in a randomized trial. Both are given by intravenous infusion every two weeks. They are available in Europe and in many other parts of the world, but treatment costs remain very high.Pharmacological chaperone therapy is another strategy to maintain enzyme activity. It does so by assisting correct folding of alpha-galactosidase despite the mutations that cause Fabry disease. As of March 2022, one medical drug based on pharmacological chaperone therapy is available for Fabry disease:
Migalastat, sold under the brand name Galafold by the company Amicus Therapeutics, is a pharmacological chaperone that can stabilize many mutant forms of alpha-galactosidase. It is taken by mouth. In a randomized trial comparing Migalastat with enzyme replacement therapy, the efficacy and safety of both treatments were similar. The US Food and Drug Administration (FDA) granted Galafold orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agencys Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016. FDA approval followed in 2018.
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. The genetic mutation that causes Fabry disease interferes with the function of an enzyme that processes biomolecules known as sphingolipids, leading to these substances building up in the walls of blood vessels and other organs. It is  inherited in an X-linked manner. Fabry disease is sometimes diagnosed using a blood test that measures the activity of the affected enzyme called alpha-galactosidase, but genetic testing is also sometimes used, particularly in females. The treatment for Fabry disease varies depending on the organs affected by the condition, and the underlying cause can be addressed by replacing the enzyme that is lacking. The first descriptions of the condition were made simultaneously by  dermatologist Johannes Fabry and the surgeon William Anderson in 1898. Signs and symptoms
Symptoms are typically first experienced in early childhood and can be very difficult to diagnose; the rarity of Fabry disease to many clinicians sometimes leads to misdiagnoses. Manifestations of the disease usually increase in number and severity as an individual ages. Pain
Full-body or localized pain to the extremities (known as acroparesthesia) or gastrointestinal (GI) tract is common in patients with Fabry disease. This pain can increase over time. This acroparesthesia is believed to be related to the damage of peripheral nerve fibers that transmit pain.