Acute Hepatic Porphyria

LONDON — Long-term givosiran treatment provided sustained benefit and improved quality of life among patients with acute hepatic porphyria, according to research presented at the International Liver Congress.

Alnylam Pharmaceuticals Inc. reported new data from the open-label extension period of the Envision phase 3 study that further demonstrated the long-term therapeutic benefit of Givlaari in patients with acute hepatic porphyria.

Patients with acute hepatic porphyria treated with Givlaari experienced a lower rate of porphyria attacks, according to results of the ENVISION study.

Alnylam Pharmaceuticals announced a new and enhanced framework for value-based agreements designed to provide access to the recently FDA-approved Givlaari for patients with acute hepatic porphyria, according to a press release.

The FDA approved Givlaari for the treatment of adults with acute hepatic porphyria, a rare genetic disorder that leads to buildup of toxic porphyrin molecules that form during the production of heme.

Results from the EXPLORE prospective natural history study designed to characterize disease activity and clinical management in patients with acute hepatic porphyria, or AHP, who experience recurrent attacks showed that attacks often required treatment in health care facilities and influenced day-to-day functioning.