Potential new drug for disease causing dilated cardiomyopathy gets special status

23rd January 2017

A proposed new drug to treat a disease that often leads to dilated cardiomyopathy has been given special status by the European Commission.

The oral drug, currently called PBI-4050, has been granted organ drug designation for the treatment of Alström syndrome (AS), a very rare disease that causes fibrosis in organs including the heart, liver and kidneys. Around half of children with the disease develop severe dilated cardiomyopathy within the first few weeks of life.

Dilated cardiomyopathy can also recur or develop for the first time in more than six in ten young people or adults with AS.

The drug’s developer, Canadian based ProMetic Life Sciences, is currently doing early trials of the drug in the UK to see the effects on vital organs in patients with AS.

Dr. John Moran, the company’s chief medical officer, said: “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 added: "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."

The European Medicines Agency had decided that the intention to treat AS with PBI-4050 was justified based on early clinical data showing an improvement in liver fibrosis. 

European orphan drug designation is granted to novel drugs that treat a rare disease or condition affecting fewer than 250,000 patients in the European Union. The designation provides the developer with various marketing and regulatory advantages.

For more details, see here.