Alnylam's highly innovative drug Givlaari is approved by the EU

Alnylam Pharmaceuticals is an industry-leading RNAi therapy company whose drug Onpattro (patisiran) was approved by the United States and the European Union in August 2018 for hereditary ATTR (hATTR) amyloidosis adult patients with stage 1 or stage 2 multiplex Treatment of neuropathy. With this approval, Onpattro became the first RNAi drug approved for marketing in 20 years since the discovery of the RNAi phenomenon.

Recently, the company announced that the European Commission (EC) has approved its other RNAi drug Givlaari (givosiran), which is administered by subcutaneous injection for the treatment of acute hepatic porphyria (AHP) aged 12 years and over Juvenile and adult patients. In the United States, Givlaari has received FDA approval for the treatment of adult patients with AHP in November 2019. It is worth mentioning that Givlaari is the first and only treatment proven to prevent AHP attacks, reduce chronic pain, and improve the quality of life. The results from the Phase III ENVISION study showed that Givlaari significantly reduced the incidence of porphyria by 74%.

Givlaari is the second RNAi drug in the world that has received regulatory approval from Alnylam, and the first global approval of GalNAc-coupled RNA therapy, marking a major milestone in the development of precision genetic drugs. In the United States, Givlaari was approved through the FDA ’s priority review process and has previously been granted breakthrough drug qualifications and orphan drug qualifications. On the EU side, Givlaari has been approved through an accelerated evaluation process and has previously been granted priority drug qualifications (PRIME) and orphan drug qualifications.

Givlaari is developed using Alnylam's enhanced and stable chemical ESC-GalNAc conjugate technology, which can make subcutaneous administration more potent and durable, and has a broad therapeutic index. John Maraganore, CEO of Alnylam, said: "The approval of Civlaari is a historic moment for patients and families with this devastating genetic disease. We are proud to release the second RNAi within 18 months The drug is brought to patients in Europe. "