Author Archives: Patricia Inacio PhD

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.

Two Previously Unknown Mutations Identified in Rare Case of Harderoporphyria

Researchers identified two previously unknown mutations in the coproporphyrinogen oxidase (CPOX) gene as the underlying cause of a rare form of hereditary coproporphyria, called harderoporphyria, in a 78-year old man. The study, “Harderoporphyria: Case of lifelong photosensitivity associated with T compound heterozygous coproporphyrinogen oxidase (CPOX) mutations“ was published in the…

Givosiran Cuts Rate of Attacks in Acute Hepatic Porphyria, Trial Shows

Alnylam Pharmaceuticals has announced that its investigational therapy givosiran achieved the primary efficacy and the majority of secondary endpoints in the ENVISION Phase 3 trial with acute hepatic porphyria patients. Treatment with givosiran significantly reduced the annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visits, or hemin administration at home…