RESEARCH TRIANGLE PARK, N.C. – BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that 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 hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
“HAE UK welcomes the decision from the MHRA to grant marketing authorization for berotralstat in the UK. Hereditary angioedema is an unpredictable and life-threatening condition which causes significant emotional and economic burdens on people with HAE and their families and careers. These burdens negatively impact their mental health and well-being, on top of the physical demands of living with a chronic condition. 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,” said Laura Szutowicz, chief executive officer 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,” said 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,” said Jon Stonehouse, president and chief executive officer of BioCryst.
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).
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.
The UK Summary of Product Characteristics (SPC) and Patient Information Leaflet (PIL) for ORLADEYO will be available from the MHRA website at https://products.mhra.gov.uk/.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States, European Union, Japan and the United Kingdom for the prevention of HAE attacks in adults and pediatric patients 12 years and older. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company’s website at www.biocryst.com.
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