NICE nod for gene silencing treatment to treat life-threatening form of porphyria

UK health technology assessor the National Institute for Heath and Care Excellence (NICE) has recommending the use of Givlaari (givosiran) on the NHS in England as an option for the treatment of acute hepatic porphyria (AHP) in people 12 years and older who have clinically confirmed severe recurrent attacks (four attacks or more within 12 months).

The approval was granted to the UK subsidiary of USA-based Alnylam Pharmaceuticals (Nasdaq: ALNY), which noted the NICE has published a Final Evaluation Determination (FED) following a Highly Specialized Technology (HST) appraisal. AHP refers to a family of ultra-rare, genetic diseases characterised by extremely painful, potentially life-threatening attacks. Some patients with AHP may experience recurrent attacks that can require periods of hospitalization, as well as ongoing chronic symptoms. Givosiran is the first and only treatment that addresses the underlying cause of this disease, the company noted.

UK pricing for Givlaari was not mentioned, but Alnylam has set a US list price of $575,000 per year, or $442,000 annually after discounts. The drug was approved by the European Commission in March 2020, and by the US regulator in 2019.