Givlaari, an injection for subcutaneous use, is an RNAi therapeutic that targets aminolevulinic acid synthase 1 (ALAS1) to treat AHP, an ultra-rare condition where patients experience severe abdominal pain, vomiting and seizures.
The EC approval was based on positive data from the ENVISION phase 3, randomised, double-blind, placebo-controlled, global and multicentre study, which assessed the efficacy and safety of givosiran in patients with a documented diagnosis of AHP.
According to the company, the reduction relative to placebo in the annualised rate of composite porphyria attacks is the primary endpoint of the trial.
Alnylam recruited 94 patients with AHP at 36 study sites in the trial in 18 countries across the world.
The givosiran significantly reduced the rate of porphyria attacks that required hospitalisations, urgent healthcare visits or intravenous hemin administration at home compared to placebo, said the company.
The European Medicines Agency (EMA) already granted priority medicines (PRIME) designation for Givlaari.
In November 2019, the company also secured approval from the US Food and Drug Administration (FDA) for Givlaari. The drug is under priority review in Brazil.
Alnylam CEO Dr John Maraganore said: “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.
“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.”
In August 2019, the investigational RNAi therapeutic givosiran has been granted priority review by the FDA for the treatment of AHP.