BioMarin's Vimizim For Morquio A Syndrome Approved By Health Canada
Biotech firm BioMarin Pharmaceutical reported that it has received approval from Health Canada for Vimizim (elosulfase alfa) for long-term enzyme replacement therapy (ERT) in patients with Morquio A syndrome.
Vimizim is an ERT that targets the deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS), the underlying cause of Morquio A syndrome. The therapy replaces inadequate enzyme activity to slow the disease’s progression. The approval makes the drug the first in line treatment option available in the country for patients afflicted with the life-limiting disease. Vimizim has also received approval in the U.S. and EU for the treatment of Morquio A syndrome.
Hank Fuchs, CMO of BioMarin, said, “The Health Canada approval of VIMIZIM is a significant milestone for BioMarin, and for Canadians living with Morquio A and their families… We will continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases.” Fuchs said that the approval of Vimizim is a significant contribution in the advancement of therapies for MPS diseases, for which the company has developed drugs for three disorders.
Morquio A syndrome, also known as a mucopolysaccharidosis IVA (MPS IVA), is caused by a lack of enzyme activity necessary to break down and remove the mucopolysaccharides, keratan sulfate. The accumulation of keratan sulfate in the cells causes progressive damage that manifests in joint abnormalities, systemic skeletal dysplasia, and short stature. It is unknown just how many people are affected by Morquio A, but estimates vary between one in 76,000 and one in 640,000. In the developed world, affected patients are estimated at 3,000.
Dr. John Mitchell, clinical investigator in the VIMIZIM Phase 3 trial, said, “Morquio A is an ultra-rare disease for which there is no cure. In fact, until now, available treatments only addressed the symptoms of the disease. The approval of VIMIZIM fills a critical unmet need for Morquio A patients and their families as it moves treatment beyond supportive care to addressing the underlying cause of the disease.”
The company filed the New Drug Submission for Vimizim with Health Canada under a Priority Review Status, which enables fast-track review of drug applications intended to treat, prevent, or diagnose serious, deadly, or severely debilitating diseases and conditions.