Purpose Pharma develops and commercializes specialty pharmaceuticals for rare diseases with high unmet medical need. The company brings innovative treatments such as ATTROGY® (diflunisal) to European markets, focusing on hereditary transthyretin-mediated amyloidosis. It also supplies unlicensed medicines to healthcare systems to expand patient access to rare‑disease therapies.
Funding
Funding not disclosed
Founders
Product
Problem
Many patients with rare diseases lack access to effective and convenient treatments, leading to significant unmet medical needs and reduced quality of life. Existing therapies may be limited, inconvenient, or ineffective in addressing the underlying causes and symptoms of these conditions. Additionally, shortages of licensed pharmaceuticals can further complicate treatment options.
Solution
Purpose Pharma focuses on the development and commercialization of innovative rare disease and specialty care products to address areas of high unmet medical need. The company develops niche products for commercialization, such as Attrogy® (diflunisal) for the treatment of transthyretin amyloidosis (ATTR), a rare and debilitating disease. Purpose Pharma also provides unlicensed medicines in certain markets to address drug shortages and engages in the export of pharmaceuticals from Europe to other countries, ensuring broader access to needed medications.
Target Audience
The primary target audience includes patients with rare diseases, particularly those with transthyretin amyloidosis, as well as physicians, pharmacies, and healthcare systems seeking specialized treatments and solutions for drug shortages.
Features
- Development and commercialization of novel therapies for rare diseases and specialty care.
- Focus on transthyretin amyloidosis (ATTR) treatment with Attrogy® (diflunisal), an oral therapy for stage 1 and 2 ATTR polyneuropathy.
- Orphan Drug Designation (ODD) for diflunisal in Europe and a pending ODD application in the US.
- Import and supply of unlicensed pharmaceutical products to address unmet medical needs when licensed options are unavailable.
- Export of pharmaceuticals from Europe to other countries to expand access to medications.
- GDP compliance certification and wholesaler distribution authorization from the Swedish Medical Products Agency.