Stockholm, July 31, 2025 – Purpose Pharma International AB today announced that the European Commission (EC) has granted ATTROGY® (diflunisal) marketing authorization for the treatment of hereditary transthyretin-mediated (hATTR or ATTRv) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
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.
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).1
• Treatment with diflunisal (a 250 mg oral tablet taken twice daily) significantly delayed disease progression (mean NIS+7 increase of 8.2 points) compared with placebo (mean NIS+7 increase of 26.3 points), resulting in an 18.0 points difference relative to placebo (p<0.001).
The baseline to 24 months change in secondary endpoints supported the impairment of disease progression demonstrated by NIS+7 scores. The Kumamoto clinical score, NIS, NIS Lower Limb (NIS-LL), and Physical Score of the Short Form (SF)-36 health-related quality of life survey demonstrated a statistically significant benefit of diflunisal treatment compared to placebo.
“We are extremely delighted that diflunisal has been approved as a therapy for patients with hereditary ATTR amyloidosis polyneuropathy. Having an effective oral treatment with a favorable safety profile that addresses the needs of a broad range of patients, represents a significant milestone for the ATTR amyloidosis community,” says Dr Jonas Wixner, Head of the Amyloidosis Centre in Umeå, Sweden.
Dr Laura Obici, Head of Rare Diseases Unit and Consultant at Amyloidosis Research and Treatment Centre at IRCCS Fondazione Policlinico San Matteo, Pavia, Italy, continues “Having been involved in the pivotal multi-national phase 3 clinical trial of diflunisal, it feels very rewarding that physicians will finally be able to prescribe diflunisal, an effective and well-tolerated oral treatment backed by a wealth of clinical experience in this disease”.
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.
About ATTROGY® (diflunisal)
ATTROGY® (diflunisal) is the first oral treatment indicated for use in hereditary ATTR amyloidosis patients with stage 1 and stage 2 polyneuropathy. Diflunisal is a difluorophenyl derivate of salicylic acid. The mode of action of diflunisal is to stabilise the TTR tetramer. Studies have shown that diflunisal binds to and stabilizes TTR in its tetramer form thereby preventing its dissociation to monomers, which is the critical step in amyloid formation.2,3,4
About Purpose Pharma
Purpose Pharma is a European pharmaceutical company based in Stockholm, Sweden, focused 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® (diflunisal) for the treatment of hereditary transthyretin-mediated amyloidosis. 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
References
1. Berk JL, Suhr OB, Obici L, et al. Repurposing Diflunisal for Familial Amyloid Polyneuropathy: A Randomized Clinical Trial. JAMA. 2013;310(24):2658–2667. doi:10.1001/jama.2013.283815
2. Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid. 2006 Dec;13(4):236-49. doi: 10.1080/13506120600960882. 3. Tojo K, Sekijima Y, Kelly JW, Ikeda S. Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res. 2006 Dec;56(4):441-9. doi: 10.1016/j.neures.2006.08.014. Epub 2006 Oct 6.
4. Koike H, Katsuno M. Ultrastructure in Transthyretin Amyloidosis: From Pathophysiology to Therapeutic Insights. Biomedicines. 2019 Feb 5;7(1):11. doi: 10.3390/biomedicines7010011.