Rare Disease Report

New Treatment for MPS VI (Maroteaux-Lamy Syndrome) In Development

AUGUST 10, 2017
James Radke
France-based biopharma company Inventiva announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to odiparcil (formerly known as IVA336) for the treatment of mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome.
MPS VI is a rare lysosomal disorder due to a reduction or absence of the enzyme arylsulfatase B that leads to toxic accumulation of glycosaminoglycans (GAGs). Common symptoms can include coarse facial features, corneal clouding, joint abnormalities and skeletal malformations, hepatosplenomegaly, and hearing loss.
Current treatment for MPS VI is enzyme replacement therapy [Naglazyme (galsulfase)] or hematopoietic stem cell transplant (HSCT) but more treatment options are needed.
Odiparcil is a substrate reduction therapy. Details of its mechanism of action are not clear, but it is believed to help synthesize a soluble form of GAGs that can be eliminated via urine secretion and thereby reduce the intracellular pool of GAGs that is acculumated in the body of MPS VI patients. Due to its unique mechanism of action, odiparcil could also be beneficial for patients with MPS I (Hurler symdrome) and MPS II (Hunter syndrome).
At the MPS Society National Conference held July 7-9, 2017 in Coventry, England, Chris Hendriksz, M.D., of the University of Pretoria, South Africa, presented data showing that in animal models of MPS VI, odiparcil restored corneal structure in the eye and also reduced GAG accumulation in the liver, kidney, spleen, heart, eye, and skin of diseased animals. Mobility and skeletal meausurements were also improved in the animals.
A Phase 2a study is in development that should begin by the end of the 2017.
 “The FDA’s decision to award Orphan Drug Designation to odiparcil in the treatment of MPS VI patients is another important milestone as it confirms that odiparcil may bring a significant therapeutic benefit over existing approved medications,” said Pierre Broqua, Chief Scientific Officer and Co-Founder of Inventiva. “MPS VI remains a severe disease despite the availability of an enzyme replacement therapy, and this decision is a strong and encouraging signal from the FDA to move forward with our clinical program.”
Orphan Drug Designation is granted by the FDA to novel therapies being developed for a rare disease. The designation allows the drug developer to be eligible for a 7-year marketing exclusivity if the drug is approved. Furthermore, the designation provides certain tax and filing fee exemptions.

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