By Toni Clarke
Feb 14 The U.S. Food and Drug Administration
said on Friday it has approved the first drug to treat Morquio A
Syndrome, a rare genetic disorder that causes skeletal
malformation and a variety of related lung, eye, ear and heart
The drug, however, will carry a boxed warning, the most
serious possible, to include the risk of anaphylaxis, an
allergic reaction that threatened the lives of some patients
during clinical trials.
The addition of the boxed warning caused shares of BioMarin
Pharmaceutical Inc., the drug's maker, to slip 0.2
percent to $75.81 on Friday.
Approval of the drug, Vimizim, follows a positive
recommendation in November from an advisory committee to the
Morquio A Syndrome is caused by a deficiency of an enzyme
known as N-acetylgalactosamine-6-sulfatase, which causes
excessive storage in the body of long chains of sugars known as
glycosaminoglycans. The disease affects about 800 people in the
United States, the FDA said.
This build-up can lead to short stature and joint
abnormalities that limit mobility and endurance. The disease can
also cause hearing loss, eye problems and heart disease.
Symptoms often appear before the age of five.
After 24 weeks of treatment, Vimizim, also known as
elosulfase alfa, increased patients' six-minute walk distance by
22.5 meters compared with those taking a placebo. Parents told
the advisory committee meeting in November that they saw
improvements in their children's energy and endurance levels,
enabling them to better socialize, learn and function
Morquio A Syndrome is one of a group of lysosomal storage
disorders known as mucopolysaccharidoses (MPS). Lysosomal
storage disorders are typically chronic and progressive, and
involve multiple organs of the body.