by Max Verhage | Dec 9, 2025 | Blog
You’ve spent years developing a promising compound as a professor, clinician, or researcher. The preclinical data looks solid. There’s clear potential to help patients with an unmet medical need. But now you face a tough question: how do you get your...
by Hellen Houlleberghs | Nov 12, 2025 | Blog
What is a Paediatric Investigation Plan (PIP)? The European Medicines Agency (EMA) requires sponsors to follow Paediatric Investigation Plan (PIP) guidelines as part of the regulatory process for developing medicines intended for children. While other regulatory...
by Max Verhage | Oct 6, 2025 | Blog
Cystinosis is a rare hereditary disease which leads to accumulation of cystine in the lysosomes causing organ failure. The disease manifests in all organs, but the kidneys and the eyes are affected first. Even though for cystinosis there is a therapy available,...
by Hellen Houlleberghs | Sep 12, 2025 | Blog
Investigator-initiated clinical trials trials play an important role in advancing medical knowledge, exploring novel uses for existing therapies, and addressing clinically relevant questions that industry trials may overlook. However, these trials come with unique...
by Ivo De Nooijer | Jul 14, 2025 | Blog
Repurposing existing drugs for new indications has emerged as a powerful strategy in modern drug development. Yet, the business models that underpin successful repurposing ventures remain diverse and highly dependent on how intellectual property (IP) is managed. In...
by Hellen Houlleberghs | Jun 17, 2025 | Blog
A promising rare disease treatment supported by Orfenix. At Orfenix, we are dedicated to turning academic breakthroughs into life-changing therapies through efficient and socially responsible drug development. One of our key projects focuses on guanabenz, a promising...
