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HomeLatest Pharma-NewsNICE Recommends BioCryst’s ORLADEYO® to Prevent Attacks in HAE Patients

NICE Recommends BioCryst’s ORLADEYO® to Prevent Attacks in HAE Patients

Sept. 15, 2021: “BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE) has recommended ORLADEYO® (berotralstat) for preventing recurrent attacks of hereditary angioedema (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.

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“HAE is a very rare genetic condition which is at best painful and debilitating, and can be fatal if left untreated. The unpredictability of the condition severely affects quality of life for patients and their families,” said Laura Szutowicz, chief executive officer of HAE UK.

“HAE UK welcomes the NICE decision on berotralstat, which means that eligible patients and clinicians have another choice of treatment for this lifelong condition.”

“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,” said Charlie Gayer, chief commercial officer of BioCryst.

The decision follows European Commission (EC) marketing authorization of ORLADEYO in April 2021 and approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in May 2021.

A decision from the Scottish Medicines Consortium (SMC) for use of ORLADEYO for HAE patients in Scotland under the UK’s National Health Service (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, as demonstrated in long-term data presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2021.

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,” said 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.”

The UK Summary of Product Characteristics (SPC) and Patient Information Leaflet (PIL) for ORLADEYO are available from the MHRA website at

About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. 

One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.”


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