The US Food and Drug Administration (FDA) has approved the sale of the Galafold drug. It is the first drug taken orally against Fabry disease. Galafold is specifically indicated for adults with a specific genetic abnormality. The drug replaces the missing enzyme, but acts differently from normal replacement therapies.
Until now, treatments for Fabry's Disease included the replacement of the enzyme alpha-galactosidase A. The new drug, on the other hand, binds to the unstable forms of the enzyme and stabilizes them. The process ensures that the recovered enzyme can be transported where it is needed. The tests confirmed the effectiveness of this approach.
The researchers performed a total of 4 clinical trials, analyzing the responses to Galafold of 139 patients. The last clinical trial was used to test the efficacy of the drug and involved 45 adults with Fabry disease. Half of the patients received the drug for 6 months, the others received a placebo.
The first group showed a clear reduction of globotriaosylceramide in the blood vessels of the kidneys. The most significant side effects were headache, nausea, urinary tract infections and above-average body temperature. However, being much less incisive than the benefits, the drug has been approved for human use.
For Galafold, the rapid approval procedure was used, which was used for the most serious diseases. Nevertheless, further studies will be needed to identify any long-term side effects.