News

MT-7117 Poised for Phase 3 Trial in Two Types of Porphyria

A Phase 3 clinical trial of Mitsubishi Tanabe’s investigational therapy MT-7117 (dersimelagon) for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) has been launched. The study (NCT04402489) will take place at 12 clinical sites in the U.S. and will test the therapy in adults and adolescents.

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

Scenesse Launched in China for Treating EPP Patients

Scenesse (afamelanotide) will be made available in China for people with erythropoietic protoporphyria (EPP), following the partnership between the treatment’s developer, Clinuvel, and the Chinese company Winhealth Pharma. The companies will collaborate to provide Scenesse — the first approved treatment for EPP — to large…