Clovertex Target of the Month (12/25): alpha-galactosidase

Clovertex’s January Target of the Month is alpha-galactosidase A, a lysosomal enzyme whose deficiency leads to Fabry disease, a rare genetic disorder that causes harmful lipid accumulation and progressive organ damage. This focus follows BioMarin’s planned $4.8 billion acquisition of Amicus Therapeutics, the company behind Galafold®, an oral therapy designed to enhance the function of specific mutant forms of alpha-galactosidase A.

Under normal conditions, alpha-galactosidase A breaks down a lipid called globotriaosylceramide (GL-3) inside lysosomes. Mutations in the GLA gene can impair this process, resulting in the toxic buildup of GL-3 in organs such as the heart, kidneys, and nervous system. Galafold® (migalastat) binds to certain unstable forms of the enzyme, helping them fold properly and reach the lysosome where they can work effectively. This targeted approach allows eligible patients to benefit from a personalized, oral treatment that supports their own enzyme function.