The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for oral, once-daily Orladeyo (berotralstat) for the routine prevention of recurrent HAE attacks in HAE patients 12 years and older.
“HAE UK welcomes the decision as HAE is an unpredictable and life-threatening condition which causes significant emotional and economic burdens on people with HAE and their families and careers. An oral therapy that shows effective control of HAE attacks will provide a different treatment choice for clinicians and patients and will assist in improving the quality of life of those living with the condition,” says Laura Szutowicz, CEO of HAE UK.
“This is a significant milestone in furthering our ability to meet the needs of more patients living with HAE. As the first oral, once-daily therapy proven to reduce the number of HAE attacks, today’s news has the potential to offer a convenient new treatment option to improve the lives and outcomes of patients with HAE,” says Dr. Sorena Kiani, consultant immunologist at Barts Health NHS Trust.
A decision from the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for use of Orladeyo under the UK’s National Health Service (NHS) is anticipated in the fourth quarter of 2021.
“With the approval of the first oral, once-daily treatment in the UK, BioCryst continues to bring Orladeyo to HAE patients and their families around the world,” says Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc.
In the pivotal Phase 3 APeX-2 trial, Orladeyo significantly reduced attacks at 24 weeks, and this reduction was sustained through 48 weeks. HAE patients who completed 48 weeks of treatment (150 mg) saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy. In the long-term open label APeX-S trial, patients completing 48 weeks of therapy (150 mg) had a mean attack rate of 0.8 attacks per month.
Orladeyo was safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving Orladeyo compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with Orladeyo, became less frequent with time and typically self-resolved.
HAE patients note a significant treatment burden associated with existing prophylactic therapy. In addition to reducing HAE attack rate, data from APeX-2 show that patients reported meaningful improvements in both quality of life, overall patient-reported satisfaction, and significant reductions in their monthly use of standard of care on-demand medicine, while taking oral, once-daily Orladeyo (150 mg).