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.
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.
Contact Orfenix.
For more information or to explore partnership opportunities, feel free to reach out to us anytime!
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