Joana Carvalho, PhD, managing science editor —

Alnylam Pharmaceuticals announced new findings from several clinical trials that continue to demonstrate the effectiveness of givosiran, the company’s investigational therapy for the treatment of acute hepatic porphyria (AHP). The findings were presented at the 2019 International Congress on Porphyrins and Porphyrias (ICPP), held Sept. 8-11…

Glycyrrhizin may be an effective treatment option for patients who develop porphyria cutanea tarda associated with alcohol abuse, a case report says. The case report study, “A first report of porphyria cutanea tarda successfully treated with glycyrrhizin,” was published in the journal of Dermatologic Therapy. Porphyria cutanea…

People with porphyria cutanea tarda (PCT) have higher risks of developing gastrointestinal diseases and certain types of cancer, compared with individuals from the general population, a study says. They also are more likely to die prematurely. The findings of the study, “Increased mortality in patients with porphyria…

Continued support, counseling, and education are important for patients with acute intermittent porphyria so that they can use that knowledge to avoid disease recurrence, a study says. The findings of the study, “Self-efficacy and self-management strategies in acute intermittent porphyria,” were published in BMC Health Services Research.

Non-invasive bioenergetic tests may be useful biomarkers to assess disease activity in patients with different types of porphyria, a study says. The study, “Feasibility of cellular bioenergetics as a biomarker in porphyria patients,” was published in Molecular Genetics and Metabolism Reports. Porphyrias are a group of genetic…

Alnylam Pharmaceuticals has completed its submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA), seeking the approval of givosiran for the treatment of acute hepatic porphyria (AHP). Givosiran, also known as ALN-AS1, is an investigational RNA-based therapy designed to…

Patients with porphyria cutanea tarda have higher risks of developing liver and other types of cancer and of dying prematurely compared to individuals from the general population, a study says. The findings of the study, “Porphyria cutanea tarda increases risk of hepatocellular carcinoma and premature death: a nationwide…

Prophylactic (preventive) treatment with heme reduces the frequency of acute attacks, improving the quality of life and reducing healthcare costs of patients with acute intermittent porphyria, a study has found. The study, “Benefits of prophylactic heme therapy in severe acute intermittent porphyria,” was published in Molecular Genetics and…

A Phase 3 clinical trial evaluating the effects of givosiran, an investigational therapy for acute hepatic porphyria (AHP), has completed patient enrollment, Alnylam Pharmaceuticals announced. The trial (NCT03338816), called ENVISION, enrolled a total of 94 AHP patients at 36 sites in 18 countries, ahead of schedule and surpassing the…