FDA approved Galafold (migalastat) as the first oral medication for the treatment of Fabry disease that is a rare genetic disorder.
Fabry disease is a rare and inherited genetic disorder when a type of fat called Globotriaosylceramide is built (GL-3) in blood vessels, the kidneys, the heart, the nerves and other parts of the body & it affects both females & males.
Due to the researches, the most severe type of this disorder called” classic Fabry disease” that affects approximately one in 40,000 males. Another type which is more frequent , occurs in one in 1,500 to 4,000 males. There are some symptoms such as slowly progressive kidney disease, cardiac hypertrophy (enlargement of the heart), arrhythmias (abnormal heart rhythm), stroke and early death. The efficacy of this medication was confirmed & they examined it on 45 adults, however there are some side effects that bothers the patients like; headache, nasal and throat irritation (nasopharyngitis), urinary tract infection, nausea, and fever (pyrexia).
Nowadays there is an unmet medical need for certain diseases like fabry diseases, in this case a further study is required to clarify the clinical benefits of Galafold. Furthermore it also received orphan drug designation as a medication for a rare disease.