According to a story from globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is to be held from February 10th-13th, 2020. This trial tested the drug odiparcil both on its own and alongside enzyme replacement therapy as a treatment for Maroteaux-Lamy syndrome. This trial produced positive results. Inventiva is focused on developing treatments for lysosomal storage disorders, fibrosis, and cancer.
About Maroteaux-Lamy Syndrome
Maroteaux-Lamy syndrome, which is also referred to as mucopolysaccharidosis VI (MPS VI), is a rare lysosomal storage disease. The effects usually appear at birth. The disease is linked to a deficiency of an enzyme called ASRB. This enzyme normally breaks down chondroitin sulfate and dermatan sulfate. The deficiency is caused be a genetic mutation that is inherited in an autosomal recessive pattern. The disease can cause a range of serious symptoms which can vary between patients. They include nerve pain, cloudy corneas, deafness, delayed walking, growth inhibition, hernias, heart disease, limited movement, skeletal deformities, macrocephaly, and enlarged liver and spleen. Unlike some related illnesses, intelligence is often unaffected. Treatment may include enzyme replacement therapy, certain medications for heart problems or infections, and various surgical procedures. Disease progression and severity range widely as does life expectancy; airway obstructions or heart problems are the most common causes of death for patients with Maroteaux-Lamy syndrome. To learn more about Maroteaux-Lamy syndrome, click here.
About The Study
The study was conducted over a 26 week period and took place at four treatment sites in Europe. 20 patients aged 16 years or older were enrolled for the study. The primary endpoint for this trial was maintaining patient safety, whereas secondary endpoints were focused on efficacy and included improvements in the function of the lungs, eyes, ears, and cardiovascular system; changes to mobility and activity levels; and changes in glycosaminoglycan (GAG) content in the skin, urine, and leukocytes. Patients that were already being treated with enzyme replacement therapy received odiparcil in addition to their current treatment.
Odiparcil has earned orphan designations in Europe and the US as well as Rare Pediatric Disease designation in the US for Maroteaux-Lamy syndrome and there are also plans to develop it for other related diseases.