Print“Vimizim is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families, says Jean-Jacques Bienaimé, CEO of BioMarin.”
The U.S. Food and Drug Administration has approved BioMarin Pharmaceutical's Vimizim, the first FDA-approved treatment for a rare, autosomal recessive lysosomal storage disease known as Morquio A syndrome.
Vimizim, BioMarin's fifth marketed product, is intended to replace an enzyme deficiency that causes the disease. Absence of the enzyme leads to problems with bone development, growth, and mobility. There are approximately 800 patients with Morquio A syndrome in the United States, according to the FDA, and about 3,000 worldwide, says BioMarin. Prior to the Vimizim approval, patients with the rare disease had no approved drug treatment options.
"Vimizim is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families," says Jean-Jacques Bienaimé, CEO of BioMarin.
Vimizim received a priority review from the FDA and was the first drug to receive a rare pediatric disease priority review voucher from the agency, a voucher it can use to gain priority review for a future drug or sell to another company for use in a future new drug application. That program, created as part of the fifth incarnation of the Prescription Drug User Fee Act, aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
Andrew Mulberg, deputy director of the FDA's Division of Gastroenterology and Inborn Errors Products, says that the agency’s approval of the drug and granting of the rare pediatric disease priority review voucher “underscores the agency’s commitment to making treatments available to patients with rare diseases.”
Vimizim's approval was based on a clinical trial involving 176 patients with Morquio A syndrome, ranging in age from 5 to 57 years. Patients treated with the therapy showed greater improvement in a six minute walking distance test than participants treated with a placebo. On average, patients treated with Vimizim in the trial walked 74 feet (22.5 meters) farther in 6 minutes compared to the patients who received placebo.
The most common side effects experienced by patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue, effects similar to those observed in clinical trials of other enzyme replacement therapies, BioMarin says.
February 16, 2014
http://www.burrillreport.com/article-fda_approves_biomarins_vimizim_for_enzyme_disorder.html
.gif)
