LAVAL, QC- The European Commission is granting ProMetic Life Sciences lead drug candidate, PBI-4050, an orphan drug designation as a treatment for Alström Syndrome (AS).
The designation is based on 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 shief medical officer.
AS leads to severe fibrosis in vital organs, including the heart, liver, and kidneys, with progressive multi organ failure. Dr. Moran says that 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,” he said.
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.