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.
How to Select the Right Academic Development and Manufacturing Support
Choosing the wrong development manufacturing partner can derail academic research with failed submissions and costly delays. Use these practical guidelines to choose the right development partner.
Paediatric Investigation Plan (PIP) Guidelines: What, Why, When, and Who
Paediatric Investigation Plans (PIPs) are mandatory regulatory documents that ensure medicines are properly tested in children before approval. Introduced by the EU’s 2006 Paediatric Regulation, PIPs outline the timing, design, and objectives of paediatric studies, requiring collaboration between regulatory affairs, clinical teams, and medical experts. Companies must secure EMA agreement on their PIP before Phase III trials, with the review process taking up to a year. This strategic tool transforms regulatory compliance into a pathway for developing safer, more effective treatments specifically designed for paediatric patients.
Cystinosis: the past, present and future
Cystinosis is a rare hereditary disease that affects multiple organs, of which the kidneys and eyes are affected first. In conversation with Irene Kinds from the Cystinosis Patient Organisation Vlaanderen and The Netherlands, and mother of a 16-year-old living with cystinosis, we discuss what daily life with the disease looks like, why early diagnosis and adherence to therapy matter, and how new treatments could change the future for patients worldwide.
Things to get right when conducting investigator-initiated clinical trials
Investigator-initiated trials drive innovation but require careful planning. Success depends on clear protocols, compliance, data quality, safety, and transparency. This ensures credible, clinically significant results that complement industry research and advance patient care.
How does a public-private-partnerships in drug development work?
Developing a new medicine is a long, costly journey—especially in rare diseases or drug repurposing—where traditional funding models often fall short. Public-Private Partnerships (PPPs) offer a powerful alternative, bringing academics, industry, and patient groups together to share risks, combine expertise, and keep patient access at the heart of development.
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.
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