Stockholm, April 28, 2025 – Purpose Pharma International AB today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Attrogy® (diflunisal) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
The European Commission (EC) will make a legally binding decision based on the EMA recommendation within 67 days. If granted by the EC, the marketing authorization is valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Lichtenstein and Norway. Diflunisal has been granted Orphan Drug Designation (ODD) for the treatment of ATTR amyloidosis in the EU. An application for ODD in the US has been submitted.
“We started Purpose Pharma with the objective to make diflunisal available for patients suffering from ATTR polyneuropathy, a severe and debilitating disease where, despite available therapies, significant unmet needs exist. If approved by the EC, Attrogy® will be the first oral therapy approved for the treatment of both stage 1 and 2 ATTR polyneuropathy,” says Jonas Hansson, CEO of Purpose Pharma.
Hereditary transthyretin-mediated amyloidosis (hATTR) is a potentially fatal condition caused by misfolded proteins accumulating into amyloid deposits in various tissues (e.g. peripheral nerves, heart and kidneys). The disease worsens over time, and while current therapies exist, many patients still lack effective and convenient treatment options.
About Purpose Pharma
Purpose Pharma is a European based pharmaceutical company focusing on the development and commercialization of novel rare disease and specialty care products in areas of high unmet medical need. Purpose Pharma is organized in two business areas. In the Specialty/Proprietary Products business area, it is the company’s mission to identify and develop niche products for commercialization. The company’s first product is Attrogy® for the treatment of transthyretin amyloidosis, a rare and debilitating disease and for which an orphan drug designation has been granted. The Unlicensed Medicines & Exports business area focuses on providing unlicensed medicines in certain markets, addressing drug shortages as well as engaging in exports of pharmaceuticals from Europe to other countries.
For more information about Purpose Pharma, visit purposepharma.com and/or contact Jonas Hansson, CEO: jonas.hansson@purposepharma.com