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RNA Interference Therapy "Givosiran" Shows Promise for Treatment of Porphyria

Accepted submission by takyon at 2019-04-15 15:21:35

Promising Results with Givosiran in Acute Hepatic Porphyrias []

Investigational givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks versus placebo in patients with acute hepatic porphyria (AHP), researchers said here.

In the interim analysis of the phase III ENVISION trial, patients treated with givosiran experienced a mean composite annualized rate of 3.2 attacks versus 12.5 attacks in patients on placebo (P<0.0001), for a mean reduction of 74%, said Manisha Balwani, MD, of the Mount Sinai School of Medicine in New York City, and colleagues.

"We saw a robust treatment effect," said Balwani at a press conference at the European Association for the Study of the Liver (EASL) [] annual meeting.

"Currently, there are no approved therapies aimed at preventing the painful, often incapacitating attacks, and chronic symptoms associated with acute hepatic porphyria," she added. "The results from ENVISION are promising and demonstrate a strong treatment effect for givosiran, with reduction of attacks and improvement in patient-reported measures of overall health status and quality of life."

Porphyria [].

BBC has a dumbed down article: Gene-silencing: 'New class' of medicine reverses disease porphyria []

Original Submission