"Givosiran Offers Hope for Acute Hepatic Porphyria Recurrent Attacks" - Katrina Altersitz
An “exciting, novel RNAi therapy” significantly lowered acute hepatic porphyria recurrent attacks, according to data presented during the International Liver Congress 2019. “Fifty percent of the givosiran patients were attack-free for the 6-month study duration compared to 16.3 percent of the placebo-treated patients. This is a highly clinical endpoint and these patients were severely affected at baseline,” said author Manisha Balwani, MD, MS, associate professor of medicine, genetics and genomic sciences at the Icahn School of Medicine at Mount Sinai. Dr. Balwani reported that after givosiran, patients had a 74 percent lower mean annualized rate of composite attacks compared with placebo while the median annualized rate of composite attacks decreased by 90 percent relative to placebo.
— Manisha Balwani, MD, MS, Associate Professor, Medicine, Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai
Additional Coverage: MedPage Today