FDA approves Vimizim used to treat rare genetic disorder in children

The Food and Drug Administration has approved the drug Vimizim (elosulfase alfa) to treat the rare disorder Mucopolysaccharidosis Type IVA, or Morquio A syndrome.

Vimizim, produced by BioMarin Pharmaceutical Inc, is the first and only treatment for patients with the extremely rare genetic condition.

Morquio A syndrome is caused by a deficiency of a metabolic enzyme known as GALNS, and can lead to diminished functional capacity, impaired quality of life and early death. The most common signs of the disease are skeletal dysplasia, the need for frequent surgical procedures related to musculoskeletal or respiratory problems, and significant limitations in mobility.

The disease kills nearly 800 people in the United States every year and more than 3,000 people in developed countries, according to the FDA.

The effectiveness of Vimizim, which is designed to replace the missing enzyme, has been clinically tested by the FDA on 176 patients between the ages of 5 and 57. The most common side effects included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue. However, the effects of the medicine have not been tested yet on children younger than 5.

"We are thrilled that patients with Morquio A syndrome will have access to this potentially life-changing therapy and appreciate BioMarin's commitment to the MPS community and the individuals and their families who are affected by these devastating conditions," Barbara Wedehase, executive director of the National MPS Society, said in a statement.

The FDA report also recommends that packaging labels include the warning that the drug may cause anaphylaxis, an allergic-like reaction, which can be life-threatening.

The California-based pharmaceutical company has also submitted marketing applications in other countries, including the European Union, Brazil, Australia, Canada and Mexico.