Bringing medicine back to life?
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What We Do.
We turn great ideas into accessible medicines. To do that we work with academics to turn their ideas into reality and in a societally responsible manner. For us, this means building a sustainable business while ensuring patient access. Explore our portfolio to see how we realize that ambition.
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Reformulating For Cystinosis

Repurposing For PXE

Repurposing For VWM
Academic Partners.




Insights.
Should I use an existing (generic) medicinal product or develop a new one for a repurposed indication?
Drug repurposing – also called drug repositioning or drug reprofiling – is the identification of new therapeutic uses for existing medicinal products.
Challenges in Clinical Trials for Orphan Drugs
Orphan drugs, which are developed to treat rare diseases, face unique challenges in clinical trials. While the incentives for developing orphan drugs, such as tax credits and market exclusivity, have encouraged pharmaceutical companies to invest in this area, the clinical trial process remains fraught with difficulties.
Mastering EMA Briefing Documents: Essential Tips and Tricks
The European Medicines Agency (EMA) scientific advice procedure is designed to enhance communication between sponsors and regulators, streamlining the development of high-quality medicinal products. Preparing a clear and well-structured briefing document is key to obtaining meaningful feedback.
Contact Orfenix.
For more information or to explore partnership opportunities, feel free to reach out to us anytime!
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