New Drugs: Agalsidase Alfa
October 2004
in “
Australian Prescriber
”
TLDR Agalsidase alfa helps treat Fabry's disease but needs more research for long-term benefits.
Agalsidase alfa, a recombinant form of a-galactosidase A, was approved in Australia for treating Fabry's disease. In a six-month trial with 26 men, it increased normal glomeruli in renal biopsies by 21%, compared to a 27% decrease in the placebo group, and reduced urinary globotriaosylceramide levels. Although infusion reactions were common, the treatment resulted in small but significant reductions in neuropathic pain, allowing some patients to stop using analgesics. However, the long-term clinical benefits and cost-effectiveness of agalsidase alfa were uncertain, necessitating further research to determine its optimal use.
