SAN RAFAEL, Calif. (AP) — BioMarin Pharmaceutical Inc. said Tuesday that a U.S. Food and Drug Administration advisory committee has voted in favor of its drug Vimizim for the treatment of Morquio A syndrome.
Vimizim is designed to treat a rare disease called mucopolysaccharidosis Type IVA, or Morquio A Syndrome. It is a genetic disease in which the body does not produce enough of a certain enzyme, which ultimately leads to the buildup of compound called keratan sulfate in organs and tissues. That causes skeletal and joint problems and shortness, and can also cause hearing loss, clouded vision and heart valve disease.
BioMarin estimates the severely debilitating disease affects an estimated 3,000 patients in the developed world.
The company said that Endocrinologic and Metabolic Drugs Advisory Committee voted in favor of approval of Vimizim for the treatment the syndrome. While the FDA is not bound by the recommendations, it takes them into account during their review.
BioMarin's has other drugs to treat two other types of mucopolysaccharidosis
Shares of the San Rafael, Calif.-based company jumped 5 percent to $70.30 in after-hours trading following the announcement.