The United Kingdom National Institute for Health and Care Excellence (NICE) recommends Orladeyo (berotralstat) for preventing recurrent attacks of HAE in eligible patients 12 years and older if they have at least two attacks per month. With this recommendation, HAE patients in England, Wales and Northern Ireland will have access to the first oral, once-daily therapy for routine prevention of recurrent HAE attacks.
“HAE UK welcomes the NICE decision on berotralstat, which means that eligible patients and clinicians have another choice of treatment for this lifelong condition”, says Laura Szutowicz, CEO of HAE UK.
“We are excited for HAE patients that this recommendation from NICE provides access to the first oral, once-daily treatment for UK patients to achieve symptom control and experience relief from the burdens of HAE. The positive NICE recommendation also expands access to modern prophylaxis with Orladeyo, compared to the attack frequency requirements from NICE for injectable options,” says Charlie Gayer, CCO of BioCryst Pharmaceuticals, Inc.
A decision from the Scottish Medicines Consortium for use of Orladeyo for HAE patients in Scotland under NHS is anticipated in the first half of 2022.
The NICE recommendation was based on findings from the Phase 3 APeX-2 trial, in which Orladeyo met its primary endpoint, significantly reducing HAE attacks vs placebo at 24 weeks. This reduction was sustained through 96 weeks, with an 80 percent average reduction in patients’ mean attack rate per month during weeks 25-96 of the trial, compared to baseline. Orladeyo was generally well-tolerated during the treatment period with fewer drug-related adverse events reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24) and part 2 (weeks 25-48).
“The impact of HAE on patients goes beyond the potentially life-threatening swellings. It can also have a long-term effect on patients’ self-esteem and quality of life,” says Dr. Sorena Kiani, consultant immunologist at Barts Health NHS Trust: “The NICE recommendation of berotralstat is great news for clinicians and eligible patients who now have access to the first oral preventive treatment for HAE that could significantly reduce the number of attacks and may improve quality of life.”