Givlaari approved in Europe for treatment of AHP

Following a positive European Medicines advisory panel recommendation at the end of January, RNAi therapeutics company Alnylam Pharmaceuticals (Nasdaq: ALNY) yesterday 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.

This EC approval follows the recent approval of Givlaari by the US Food and Drug Administration in November 2019, with Alnylam setting a list price of $575,000 per year, or $442,000 annually after discounts. Givosiran is also awaiting approval in Brazil where it is under priority review.

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.