Alnylam Pharma Says Givlaari Approved In EU For Acute Hepatic Porphyria

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) said Tuesday that the European Commission or EC has granted marketing authorization for Givlaari, or givosiran, an injection for subcutaneous use targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria or AHP in adults and adolescents aged 12 years and older.

AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures that can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including pain, which continue to be present between attacks.

"Today's approval of Givlaari marks a historic moment for patients and families living with this devastating genetic disease, as there are currently no approved medicines in Europe proven to decrease the frequency of attacks and reduce the chronic pain that many patients suffer," said John Maraganore, Chief Executive Officer of Alnylam.

Alnylam noted that the marketing authorization for Givlaari was based on positive data from the ENVISION Phase 3 trial, a randomized, double-blind, placebo-controlled, global, multicenter study to evaluate the efficacy and safety of givosiran in patients with a documented diagnosis of AHP.

Givlaari (givosiran) was granted Priority Medicines or PRIME Designation by the European Medicines Agency (EMA) as well as Orphan Designation in the European Union. It was also granted an accelerated assessment, which is awarded to medicines deemed to be of major public health interest and therapeutic innovation, and the award is designed to bring new treatments to patients more quickly.

This EC approval follows the recent approval of Givlaari by the U.S. Food and Drug Administration in November 2019. Givosiran is also awaiting approval in Brazil, where it is under priority review.