Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the positive results of their study as MPS VI is a disease with a “highly unmet medical need.” They hope that their developments will better the lives of those with this disease.
About Mucopolysaccharidosis Type VI (MPS VI)
Mucopolysaccharidosis type VI (MPS VI) is also known as Maroteaux-Lamy syndrome. It is a rare disease that is characterized by the lack of part of or the entire arylsulfatase enzyme. Without this enzyme the body accumulates a carbohydrate called glycosaminoglycans, which impacts organ function.
Symptoms of this disease can vary from person to person. Some people may only experience mild effects while others have life-threatening symptoms. Some of these effects include vision loss, clouding of the cornea, coarse facial features, skeletal and muscular deformities, abnormalities within the joints, chronic pain that can range from mild to severe, numbness, tingling, loss of hearing, an enlarged spleen or liver, pulmonary disease, and cardiac disease.
The onset and severity of symptoms vary from person to person. At times the effects will appear during infancy and slowly develop so that they do not cause damage until adulthood. Because of this slow development it is important that if a child displays any of these symptoms that their healthcare team is notified. Severe cases of MPS VI include mobility problems, delayed or absent puberty, and heart failure in early adulthood. Milder cases with delayed progression include cardiac and pulmonary disease.
There are FDA approved treatments for this disease which must be overseen by a specialist. The first treatment was approved in 2005, and it is an enzyme replacement therapy called Naglazyme.
About Inventiva’s Study
Phase IIa of the study lasted 26 weeks and enrolled 20 people. Participants were 16 or older and had the advanced stages of MPS VI. The study was double-blind and placebo-controlled. Participants received an oral dose of either 25mg or 500mg of odiparcil or the placebo twice daily for six months. They took this drug in addition to enzyme replacement therapy, which is currently the most common treatment. 13 participants completed the study. Odiparcil showed a good safety profile, which encourages researchers to continue to the next phase of the trial.
Researchers observed that corneal clouding improved during this study, as did cardiac and respiratory functions. As adverse effects were mild to moderate, Inventiva is continuing this trial. Their target population is children, who will also be the next participants in this study.
Odiparcil has received the orphan drug designation from the FDA and European Medicines Agency (EMA). In the U.S. it has also received the rare pediatric disease designation for the treatment of MPS VI.
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