FDA grants Galafold (migalastat) 123mg hard capsule accelerated approval

recently, Amicus Therapeuticsannounced that the U.SFood andDrug(
FDA) has granted Galafold (migalastat) 123mg of accelerated approval for hard capsulesGalafold is an oral, precisionmedical(drug used in in vitroto detect(Fabry disease) in adult patients who have confirmed the presence of specific alpha-semi-lactose gene mutationsGalafold will continue to evaluate the drug as part of accelerated approval based on data that can reduce the level of nephrine capillary capillary cell ceramide triacetic sugar triglyceride (globotriaosylceramide, GL-3) through accelerated approval channel approvalGalafold's active pharmaceutical ingredient is migalastat, an oral drug "molecular companion" of alpha-semi-lactose assines A (alpha-Gal A), which selectively and reversiblely binds to specific mutation types of alpha-Gal A enzymes, stabilizing the enzymes of these dysfunctions, and facilitating their transport to the lysosome to remove accumulated disease substrates, thereby playing a therapeutic roleGlobally, it is estimated that about 35%-50% of people with Fabry disease have treatable GLA variantsGalafold has previously been approved by Australia, Canada, the European Union, Israel, Japan, South Korea and Switzerlandthis approval is based primarily on efficacy data from key Phase III clinical studies Study 011 (also known as FACETS) The study showed that Galafold significantly reduced gl-3 levels of interrenal capillaries compared to placebo safety
the most common side effects of Galafold are headache, nasopharyngitis, urinary tract infections, nausea and fever On the regulatory side, Galafold has been eligible for orphan medicine, fast-track and priority review by the FDA