Maroteaux-Lamy syndrome (MPS VI), is a rare paediatric genetic degenerative disease with an estimated prevalence of 1 in 225,000 live births.
There is no cure for MPS VI and current treatment options, such as enzyme replacement therapy (ERT) or hematopoietic stem cell transplant (HSCT), leave the patients with high unmet medical needs. Patients affected by the syndrome have coarse faces, short stature, corneal clouding, hearing loss, dysostosis multiplex, hepatosplenomegaly, cardiac valve disease and reduced pulmonary function without intellectual deficit.
Christine Lavery, President of the UK MPS Society, said: "This decision could accelerate the availability of a much needed new treatment for MPS VI patients."
Odiparcil (formerly IVA336), an orally available therapy, is being developed as a potential therapy for MPS I, II, and VI patients.
Professor Chris Hendriksz, of FYMCA Medical and University of Pretoria, South Africa, said: "Odiparcil has the potential to become a breakthrough treatment for patients affected with MPS VI and I am looking forward to the upcoming phase IIa clinical trial, which could prove the efficacy of odiparcil as a stand-alone treatment."
The European Medicines Agency (EMA) grants orphan drug designation (ODD) to support the development of medicines for the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases that affect no more than 5 in 10,000 individuals in the European Union. ODD allows companies to receive development incentives, such as protocol assistance, reduced fees for regulatory activities and up to 10 years of market exclusivity in the EU upon marketing approval for the designated indication.
Pierre Broqua, CSO and Co-Founder of Inventiva, said: "We recently received US orphan drug status and with this new EU designation we continue delivering on our regulatory strategy for odiparcil.
"Clearly the recent preclinical data we generated showing that odiparcil is active in organs where marketed enzyme replacement therapies have limited or no efficacy, has been instrumental in convincing regulators to grant these ODD designations. These designations confirm that regulatory agencies share our view that odiparcil could improve MPS VI current treatment options."
