The marketing authorization is valid in all European Union (EU) member states, as well as in the European Economic Area (EEA) countries, Iceland, Lichtenstein and Norway. 

“This is a significant milestone for Purpose Pharma as it marks the first regulatory approval for ATTROGY. We are actively pursuing market access in several European countries and are committed to making ATTROGY available to patients suffering from this severe and debilitating disease as soon as possible,” says Jonas Hansson, CEO of Purpose Pharma. 

Based on the phase 3 trial

The EC approval is based on the phase 3 randomized, double-blind, placebo-controlled trial in which treatment with the transthyretin (TTR) tetramer stabilizer diflunisal, met the primary endpoint, change from baseline at 24 months in Neuropathy Impairment Score + 7 (NIS+7).

Hereditary transthyretin-mediated amyloidosis

Hereditary transthyretin-mediated amyloidosis is a potentially fatal condition caused by misfolded proteins accumulating as amyloid deposits in various tissues and organs (e.g. peripheral nerves, heart and kidneys). The disease is progressive, and while treatment options are available, many patients still lack access to effective and convenient therapies.