FDA Approval For BioMarin's Vimizim For Morquio A Syndrome
By Cyndi Root
For a rare enzyme disorder, the Food and Drug Administration (FDA) has approved Vimizim from BioMarin, announced in a press release by the FDA. Vimizim (elosulfase alfa) is the first granted a Rare Pediatric Disease Priority Review Voucher. The drug treats Mucopolysaccharidosis Type IVA (Morquio A syndrome), a rare disease. Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research (CDER) said, “This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases.”
Mucopolysaccharidosis Type IVA
Patients with Mucopolysaccharidosis Type IVA, also known as Morquio A syndrome, had no treatment options prior to this drug approval. Morquio A syndrome is an autosomal recessive lysosomal storage disease. It is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiencies. Symptoms mainly occur in the skeleton and get progressively worse. Abnormalities usually present in childhood. People with this disease may be short, have loose joints, knock knees, and other abnormalities. Some people lose vision, while others lose hearing. Respiratory infections are common, as are facial feature abnormalities. In the United States, about 800 people have this disease.
Vimizim
Vimizim works on the metabolic pathway with the missing GALNS enzyme involved in cell growth, bone development, and joint mobility. The FDA relied on a clinical trial to prove that the drug was safe and effective. 176 people with the syndrome ranging from 5 to 57 years old took part. Walking tests were a measure of its effectiveness. Vimizim-treated patients walked about 20 meters further than those given a placebo. The medication is labeled with a warning for anaphylaxis, as some patients in the trial experienced reactions. Side effects included fatigue, abdominal pain, headache, and fever, among others.
Rare Pediatric Disease Priority Review Voucher Program
Vimizim was granted a Rare Pediatric Disease Priority Review Voucher instituted under the FDA Safety and Innovation Act (FDASIA). Under this program, the FDA expedites consideration of drugs indicated for rare, serious, and pediatric conditions. Sponsors of the drug may market the drug for the prevention and treatment of rare pediatric diseases.
About BioMarin
BioMarin develops pharmaceuticals for serious conditions. The company's product line includes several approved drugs and others in the drug development pipeline. The company plans to market and ship Vimizim immediately.
Source:
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm386008.htm