UPDATE : Tuesday, November 19, 2019
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Handok releases Fabry disease treatment
  • By Lee Han-soo
  • Published 2019.07.02 16:19
  • Updated 2019.07.02 16:19
  • comments 0

Handok said Tuesday that it has rolled out Galafold, a Fabry disease treatment developed by the U.S. pharmaceutical company Amicus Therapeutics.

Amicus Therapeutics’ Fabry disease treatment Galafold

The drug is the world's first and only oral Fabry disease treatment available for Fabry disease patients with amenable mutations.

Unlike conventional enzyme replacement therapies that inject the corresponding enzyme developed by recombinant DNA technology into the patient's body, the drug comes in a capsule form patients can take by themselves.

The drug binds to the deficient alpha-galactosidase A enzyme in the body of a Fabry disease patient with an adaptive mutation to restore enzyme activity and breaks down accumulated glycolipids.

The medicine has confirmed the efficacy and safety through a variety of clinical trials. The results of the comparative analysis of the phase 3 FACET study revealed that the galactose-containing group had a significant decrease in the accumulation of glycolipids in the kidney capillaries.

Fabry disease is a rare ailment caused by a deficiency of an enzyme called alpha-galactosidase A. The lack of the enzyme leads to the accumulation of other glycolipids, resulting in a decrease in the function of various cells in the body. The disease can cause serious complications such as cardiovascular disease, stroke, kidney failure, and even death.

"The advantage of oral treatment is that it will help reduce the difficulties of patients with Fabry disease who had to visit the hospital once every two weeks to get injections," Handok CEO Kim Young-jin said.


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