Odiparcil is a small molecule developed by Inventiva Pharma with the potential to become the first oral GAG clearance therapy for Mucopolysaccharidosis I, II, IVA, VI and VII. Odiparcil has received orphan drug designation by the FDA and EMA, rare pediatric disease designation by the FDA and successfully passed a phase IIa trial in adolescent and adult MPS VI patients.
In contrast to recombinant enzymes, which constitute the current standard of care, odiparcil can be given orally and is more widely distributed in the body. For instance distribution and exposure data generated in rodents show that odiparcil reaches the heart, joints, cartilage and cornea. Many MPS patients suffer from heart problems, stiff joints, spinal cord compression and visual impairment where enzyme replacement therapy (ERT) has limited or no efficacy. In the recent iMProveS trial in adolescent and adult MPS VI patients signs of efficacy were observed in those hard to reach tissues.
Odiparcil may offer significant therapeutic benefit to children with MPS VI. In fact, treating children at a young age may prevent the appearance of some of the most prominent symptoms in MPS VI children such as visual impairment and heart problems.
The primary objective of the Safe-KIDDS trial is to show that odiparcil can be safely administered on top of enzyme replacement therapy to MPS VI children over the age of five. In addition we are collecting pharmacodynamics (PD) and pharmacokinetics (PK) data and are looking to observe preliminary efficacy signs. If you are interested in this trial please contact one of the study investigators.