The trial results were significant, although the effects were not very large in absolute terms, and the risk-safety profile is favorable today.
Minder EI, Schneider-Yin X. · Expert Rev Clin Pharmacol (2015)
- Review of the rationale and Phase II/III results of afamelanotide (CUV1647) in protoporphyria
- Trial results were statistically significant though absolute effects were modest
- High compliance and consistent effectiveness across six years of compassionate-use in Switzerland