CHMP Recommends Marketing Authorization of First AHP Treatment

The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has recommended that Givlaari (givosiran) be granted marketing authorization in the European Union, according to a Jan. 31, 2020 press release.

Givlaari is a treatment for acute hepatic porphyria (AHP), which is a rare genetic condition and potentially life-threatening. There are no currently approved treatments that directly ameliorate or prevent chronic symptoms associated with AHP.

CHMP’s recommendation has been based on the results of a Phase III clinical study, demonstrating the benefits and safety of the treatment in 94 patients with AHP who had experienced at least two attacks within the past six months. As Givlaari addresses an unmet medical need, it has been afforded accelerated assessment by the regulatory body.

The recommendation will be submitted to the European Commission for consideration and, if approved, will be authorized for marketing across the EU for adult patients and adolescents aged 12 years and older with AHP. Authorization application for Givlaari was submitted by Alnylam Netherlands.

Source: EMA