Stockholm, November 17, 2025 – Purpose Pharma International AB today announced that ATTROGY® (diflunisal) is now available in Germany for the treatment of hereditary transthyretin-mediated (hATTR or ATTRv) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Following European Commission approval of ATTROGY® for the treatment of hereditary transthyretin-mediated amyloidosis in July 2025, ATTROGY® is now available in Germany.
“This is a next significant milestone for Purpose Pharma and hATTR patients in Germany as ATTROGY® is the first available oral treatment for adult hATTR patients covering both stage 1 and stage 2 polyneuropathy. The entire Purpose Pharma team has worked with great dedication and perseverance to make ATTROGY® available to patients suffering from this severe and debilitating disease,” says Jonas Hansson, CEO of Purpose Pharma.
Hereditary transthyretin-mediated amyloidosis is a potentially fatal condition caused by misfolded proteins accumulating as amyloid deposits in various tissues and organs (e.g. nerves, heart and kidneys). The disease is progressive and it is important to provide further treatment options and give patients access to effective and convenient therapies.
About ATTROGY® (diflunisal)
ATTROGY® (diflunisal) is the first oral treatment indicated for use in hereditary ATTR amyloidosis patients covering both stage 1 and stage 2 polyneuropathy.# Diflunisal is a difluorophenyl derivate of salicylic acid. The mode of action of diflunisal is to stabilize 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.1-3
The first study reporting diflunisal’s clinical value in ATTRv-PN patients was a multinational phase 3 randomized, double-blind, placebo-controlled trial in which treatment with diflunisal, met the primary endpoint, change from baseline at 24 months in Neuropathy Impairment Score +7 (NIS+7).4
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-point difference relative to placebo (p<0.001).
Secondary endpoint analysis confirmed impairment of disease progression. Diflunisal demonstrated statistically significant benefit over placebo in the Kumamoto clinical score, NIS, NIS-LL, and SF-36 Physical Score at 24 months.
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 Sweden to other countries.
For more information about Purpose Pharma, visit purposepharma.com and/or contact Jonas Hansson, CEO: jonas.hansson@purposepharma.com.
For medical or scientific information about hATTR amyloidosis or ATTROGY® contact medicalinfo@purposepharma.com.
References
# Attrogy SmPC
1. 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.
2. 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.
3. Koike H, Katsuno M. Ultrastructure in Transthyretin Amyloidosis: From Pathophysiology to Therapeutic Insights. Biomedicines. 2019 Feb 5;7(1):11. doi: 10.3390/biomedicines7010011.
4. 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.
Code: Corp-EU-25/0005. Date of Preparation: November 2025