FDA
21st November 2013

FDA Panel Recommends Vimizim for Approval

On Tuesday, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted in favor of approval of Vimizim for the treatment of Mucopolysaccharidosis Type IVA (MPS IVA) or Morquio A syndrome. Nineteen of the 21 panel members supported approval of Vimizim for use in all MPS IVA patients; one voted in favor of approval for a subgroup of MPS IVA patients and one panel member voted to not recommend approval. 

FDA Panel Recommends Vimizim for Approval

On Tuesday, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted in favor of approval of Vimizim for the treatment of Mucopolysaccharidosis Type IVA (MPS IVA) or Morquio A syndrome. Nineteen of the 21 panel members supported approval of Vimizim for use in all MPS IVA patients; one voted in favor of approval for a subgroup of MPS IVA patients and one panel member voted to not recommend approval. 

Vimizim (elosufase alfa) is an investigational enzyme replacement therapy (ERT) for the treatment of patients with the lysosomal storage disorder known as Morquio A syndrome. Vimizim is the first ERT designed to target the underlying cause of Morquio A Syndrome—a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Infused enzyme replacement therapy with Vimizim replaces deficient GALNS activity to minimize progressive multi-systemic manifestations. 

Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA), is an ultra-rare, severely debilitating disease that affects an estimated 3,000 patients in the developed world. The syndrome is estimated to occur in 1 of every 200,000 births, with symptoms usually beginning to show between ages 1 and 3. A family history of the syndrome raises one’s risk for the condition.

It is an inherited disease in which the body is missing or doesn’t have enough of an enzyme that is essential in the breakdown and removal of the glycosaminoglycans (GAGs) known as 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 several debilitating symptoms such as skeletal dysplasia and short stature and joint abnormalities, which limit mobility and endurance. Furthermore, 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.

“We are thrilled to have achieved this important milestone in our mission to bring the first approved therapy to treat Morquio A patients,” said Jean-Jacques Bienaimé, CEO of BioMarin. “I am very pleased with the outcome of today’s panel vote and look forward to continuing to work with the FDA to bring this much-needed therapy to these patients.”

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2014, for completion of its review of the Biologics License Application (BLA) for Vimizim. 

The FDA is not bound by the EMDAC’s recommendation, but will consider the committee’s recommendation as the FDA completes its review of the Vimizim BLA.

ISS has over a decade of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net.

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