European Union Approves Treatment for Acute Hepatic Porphyria

Alnylam Pharmaceuticals, Inc, the leading RNAi therapeutics company, announced that the European Commission (EC) has granted marketing authorization for GIVLAARI® (givosiran), an injection for subcutaneous use targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (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, which 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, Ph.D., Chief Executive Officer of Alnylam in a press release. “We are proud to bring GIVLAARI, our second RNAi medicine to be approved in the last 18 months, to patients in Europe and we want to thank the patients, families, investigators and study staff whose support and involvement have made this achievement possible.”

Read the full press release here.

Source: Alnylam Pharmaceuticals, Inc. press release