Explore Orfenix Insights, where we share our latest thoughts, research findings, and perspectives on advancing affordable therapies for rare diseases. Dive into in-depth articles, case studies, and collaborative stories that reflect our commitment to innovative drug repurposing and socially responsible healthcare.
Aligning interests: how IP can make drug repurposing work for everyone
Drug repurposing gives existing medicines a second life, but only when the business model clears the path. At Orfenix, we structure Intellectual Property strategically to align investor incentives, guarantee patient access, and deliver sustainable impact. Discover how our IP approach protects innovation and connects commercial potential with public health outcomes.
Guanabenz for Vanishing White Matter
Orfenix proudly supports the development of guanabenz for Vanishing White Matter—a rare, progressive brain disease in children. Learn how repurposed drugs and dedicated research offer hope.
Things to consider when designing a clinical trial involving children
Pediatric clinical trials are crucial for developing safe, effective treatments for children, especially those with rare diseases like VWM and cystinosis. Despite challenges, Orfenix remains committed to advancing pediatric innovation.
Understanding pseudoxanthoma elasticum: Insights from Orfenix
PXE is a rare disease with serious impact on daily life. We spoke with Heleen van Loenen from the Dutch PXE patient organisation about patient experiences—and why Orfenix is working to develop a treatment.
Exclusive to be inclusive, the ethics of exclusivity
Pharmaceutical innovation thrives on a balance between exclusivity and accessibility. Limited-time commercial exclusivity incentivizes research while ensuring broader patient access over time. Discover how these mechanisms shape the future of equitable healthcare.
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.
The Origin and Benefits of Drug Repurposing: Unlocking Potential
Drug development is a long, expensive, and risky process. It takes an average of 10-15 years and requires a multi billion investment to bring a new drug from the laboratory to patients.
Planning for access – Socially Responsible Licensing
Orfenix welcomes a recently published report of the Medicines Patent Pool, in partnership with Boston Consulting Group, shows how voluntary licensing brings both improved access to medicines, in particular in low and middle-income countries and economic benefits to the companies doing the out-licensing.
Do you want to make an impact on patients’ lives?
At Orfenix, you work with top professionals and collaborate with leading experts to bring forgotten treatments from the lab to patients.
If you’re passionate about transforming healthcare and thrive in a transparent, trust-driven environment, Orfenix is the place for you.










