BioMarin Pharmaceutical’s Vimizim has become the first drug approved in the US to treat a rare genetic disorder that affects bone development, growth and mobility.
The Food and Drug Administration (FDA) backed the drug for the treatment of Mucopolysaccharidosis Type IVA (Morquio A syndrome), an ultra rare condition which is defined by a deficiency in an enzyme called N-acetylgalactosamine-6-sulfate sulfatase (GALNS).
This enzyme plays a role in a body’s ability to metabolise glycosaminoglycan molecules, and a build of glycosaminoglycan can lead to diminished functional capacity, including severe musculoskeletal damage and respiratory dysfunction.
Vimizim (elosulfase alfa) works by mimicking the missing GALNS enzyme and has demonstrated in clinical trials its effectiveness in improving a patient’s mobility and endurance compared to placebo.
It is thought that Morquio A syndrome affects about 800 people in the US and 3,000 people worldwide, meaning it fits comfortably into the rare disease classification for the FDA.
This status provides certain incentives to company’s developing orphan drugs for rare diseases, including tax breaks.
In its filing for Vimizim, BioMarin was also able to benefit from priority review status – accelerating the appraisal process – as well as being the first company to receive a rare paediatric disease priority review voucher.
This voucher is a new initiative by the FDA which aims to aims to encourage the development of new treatments for rare diseases that affect children.
Dr Andrew Mulberg of the FDA’s Center for Drug Evaluation and Research (CDER), commented on the approval’s importance for the agency.
“This approval and rare paediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” he said.
BioMarin said it intends to begin promotion of the drug immediately in the US. Marketing applications for Vimizim are also currently under review in the EU, Brazil, Australia, Canada and Mexico.
