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VIDEO: Givosiran may ‘transform’ lives of patients with acute hepatic porphyria
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VIENNA — In this exclusive video from the 2019 International Liver Congress, Akin Akinc, PhD, of Alnylam Pharmaceuticals, discusses results of a study in which givosiran, a novel RNAi therapy, significantly lowered acute hepatic porphyria recurrent attacks.
“Unfortunately, acute hepatic porphyria can be difficult to diagnose mainly due to low disease awareness, but also do to the fact that many of the symptoms are rather non-specific,” Akinc told Healio Gastroenterology and Liver Disease. “Unfortunately, many patients go through a very long diagnostic journey that could last up to 15 years.”
Akinc noted that hopefully with the presentation of these data, as well as other efforts to increase awareness of acute hepatic porphyria, that the disease burden will be somewhat alleviated.
“If approved, givosiran has the opportunity to be transformative in the lives of patients living with AHP,” he said.
Reference:
Balwani M, et al. GS-14. Presented at: International Liver Congress; April 10-14, 2019; Vienna, Austria.
Disclosure: Akinc is an employee of Alnylam Pharmaceuticals.