To the EDITOR: The International Porphyria Patient Network welcomes additional treatments for the erythropoietic protoporphyrias, such as dersimelagon, which was described by Balwani et al. (April 13 issue).1 New drugs could help to address the needs of underserved subgroups such as children and may provide patients and health care professionals with treatment choices. The risks and benefits associated with various treatments are best assessed by applying comparable outcome measures, such as the same validated quality-of-life instruments and efficacy end points. The phase 2 trial of der-simelagon conducted by Balwani et al. missed this opportunity because its outcome measures differed from those used in the trials that assessed afamelanotide, the only approved treatment for erythropoietic protoporphyria. How ever, we hope that potential phase 3 trials will be designed with such comparable outcome measures. Moreover, dersimelagon and afamel-anotide have a similar mechanism of action (i.e., binding to the melanocortin-1 receptor MC1R of melanocytes and stimulation of eumelanin synthesis). Given that dersimelagon and afamelanotide have similar effects on skin pigmentation, we hope that phase 3, head-to-head trials will be free of unconscious bias from patients guessing correctly about the drug they are receiving and that these trials will provide a direct comparison of the safety and efficacy of these agents.
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