The US Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical's Vimizim (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

"The FDA approval of Vimizim is an important milestone for BioMarin and for patients with Morquio A syndrome. 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," said Jean-Jacques Bienaimé, chief executive officer of BioMarin. "With the approval of Vimizim, BioMarin firmly establishes its leadership in advancing therapies to treat MPS diseases. We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases."

Vimizim is an enzyme replacement treatment for Morquio A syndrome, which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance, and breathing.

"In clinical trials, Vimizim was shown to significantly improve endurance, which possibly could change the course of the disease. As a treating physician, I am encouraged that the therapy has proven to provide clinical benefit, which is not always possible to demonstrate with ultra-rare diseases," said Paul Harmatz, M.D., Associate in Gastroenterology and Nutrition at the Children's Hospital and Research Center in Oakland, California and clinical investigator in the Vimizim phase 3 trial. "The approval of Vimizim is an important advance for Morquio A patients and their families and moves treatment beyond supportive care to treating the underlying cause of the disease."

"We are thrilled that patients with Morquio A syndrome will have access to this potentially life-changing therapy and appreciate BioMarin's commitment to the MPS community and the individuals and their families who are affected by these devastating conditions," said Barbara Wedehase, MSW, CGC, executive director of the National MPS Society. "Until now, patients with Morquio A syndrome didn't have a drug treatment option. This approval provides the community with a therapy and with hope."

Shipments of Vimizim to the distribution channels will commence immediately, and BioMarin will begin promotion of Vimizim in the US immediately. BioMarin has also submitted marketing applications for Vimizim in the European Union, Brazil, Australia,  Canada, and Mexico.

BioMarin will offer support to patients through its BioMarin Patient & Physician Support (BPPS) team. Through BPPS, patients receive live, personalized support by a specialized case manager who will research insurance coverage and alternative benefit options. BPPS will help patients obtain coverage and minimize out-of-pocket expenses and find alternative financial assistance for treatment.

Vimizim (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). Vimizim is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome - a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Vimizim is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs.  Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no standard accepted treatment other than supportive care.

Morquio A syndrome, or Mucopolysaccharidosis IVA (MPS IVA) is a disease in which people are missing an enzyme that is essential in the breakdown and removal of the glycosaminoglycans (GAGs) called keratan sulfate (KS) and chondroitin-6-sulfate (C6S). The incompletely broken down GAGs remain stored in cells in the body causing progressive damage. This excessive storage causes systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.