The European Medicines Agency (“EMA”) determined that the intention to treat AS with PBI-4050 was justified based on the preliminary clinical data generated in AS patients showing an improvement in liver fibrosis.
ProMetic is currently investigating the effects of PBI-4050 on multiple organs in AS patients in an ongoing open label Phase 2 clinical study in the UK.
“The positive results in patients with AS nicely complement those already observed in our other trials in idiopathic pulmonary fibrosis and in metabolic syndrome and type 2 diabetes” commented Dr. John Moran, ProMetic’s Chief Medical Officer.
“AS leads to severe fibrosis in vital organs, including the heart, liver, and kidneys, with progressive multi organ failure. PBI-4050 has successfully reversed fibrosis in the heart, lungs, kidneys, pancreas and liver in several preclinical studies. We are very excited at the idea that PBI-4050 may well offer a real long-term benefit for these patients”.
This condition is chronically debilitating due to permanent blindness, deafness, and type 2 diabetes and life-threatening due to progressive organ failure. No satisfactory method of treatment has been authorised so far in the European Union for patients affected by AS.
Pierre Laurin, Chief Executive Officer of ProMetic commented: “We will continue to work diligently with medical experts and with the regulatory agencies to define an optimal regulatory pathway to provide a treatment for these patients affected by AS”.
Alström Syndrome is a rare inherited condition with progressive fibrosis involving multiple organs such as the liver, the kidneys and the heart. Idiopathic infantile dilated cardiomyopathy (ICM) presents acutely in nearly half of all AS subjects within the first few weeks of life.
In survivors, cardiomyopathy recurs or develops de novo in up to 65% of adolescents and adults with high rates of morbidity and mortality, at which stage coarse fibrosis is evident on post-mortem.
European Orphan Drug Designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 250,000 patients in the European Union.
The designation provides the drug developer with a ten year period of marketing exclusivity upon marketing approval for the designated indication, as well as reduced fees for regulatory activities, the ability to apply for marketing authorisation centrally in the European Union and protocol assistance, a form of scientific advice specifically for orphan medicines.