New and currently investigated pharmacotherapies for the erythropoietic protoporphyrias: spotlight on dersimelagon and bitopertin
The currently available trial data on dersimelagon and bitopertin suggests treatment effects in EPP as compared to placebo control groups. However, safety and efficacy of dersimelagon and bitopertin need to be further characterized. Moreover, to ensure benefit for patients and access to therapy after regulatory approval, it also would be important to generate data on the relative safety and efficacy as compared to afamelanotide.
Authors
- Sebastian Wäscher (ORCID: https://orcid.org/0000-0002-9657-4310)
- Rocco Falchetto (ORCID: https://orcid.org/0000-0003-3524-2141)
- Cornelia Dechant (ORCID: https://orcid.org/0000-0003-1442-3824)
- Jasmin Barman-Aksözen
- Francesca Granata
Institutions
- ETH Zurich (CH)
- Uniwersytecki Szpital Dziecięcy (PL)
- Institute for Biomedical Engineering (CH)
- Institute of Sugar Beet (CN)
- Catholic University Our Lady of Good Counsel (AL)
- Laboratoire National de Référence (MA)
- Children's Clinical University Hospital (LV)
- Innovative Research (United States) (US)
Publication Details
- Journal
- Expert Opinion on Pharmacotherapy
- Published
- 2026-03-26
- DOI
- https://doi.org/10.1080/14656566.2026.2651281
- Primary Topic
- Porphyrin Metabolism and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00