The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a favorable opinion for Attrogy, a new therapeutic option targeting hereditary transthyretin-mediated amyloidosis (ATTRv). This approval marks a significant advancement for adult patients grappling with stage 1 or 2 polyneuropathy associated with the rare condition.
Key Benefits of Attrogy in Clinical Use
Attrogy, developed by Purpose Pharma International AB, is available in 250 mg film-coated tablets. Its active ingredient, diflunisal, effectively stabilizes the transthyretin (TTR) tetramer, preventing its breakdown into monomers that lead to amyloidosis. Clinical trials demonstrated that patients treated with Attrogy experienced a notable delay in disease progression over two years compared to those on a placebo, as measured by the Neuropathy Impairment Score plus 7 nerve tests (NIS+7).
Regulatory Insights and Future Availability
The CHMP’s positive stance paves the way for the European Commission to grant marketing authorization, with detailed usage guidelines to follow in the Summary of Product Characteristics (SmPC). Once authorized, Attrogy will be accessible across all EU member states, supporting a broader patient base.
Inferences:
- Attrogy introduces a vital treatment avenue for adults with ATTRv, addressing a significant unmet medical need.
- Clinical efficacy is evidenced by the sustained delay in disease progression over a substantial treatment period.
- Minimal side effects, primarily gastrointestinal, enhance the drug’s tolerability profile.
- Orphan designation underscores the drug’s importance in treating a rare and debilitating condition.
Access to Attrogy will empower patients and healthcare providers with a new tool to manage ATTRv effectively, potentially improving quality of life and clinical outcomes. As regulatory bodies finalize approval, stakeholders anticipate broader discussions on pricing, reimbursement, and integration into existing treatment protocols. Patients currently affected by ATTRv can look forward to enhanced treatment options, while ongoing research may further elucidate diflunisal’s role in combating amyloidosis.
The approval of Attrogy exemplifies the European Union’s commitment to addressing rare diseases through dedicated regulatory pathways and supporting innovative pharmaceutical developments. This milestone not only offers hope to those impacted by ATTRv but also reinforces the importance of continued investment in treatments for rare and life-threatening conditions.
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