– Results published in JACI: In Practice show rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment with ORLADEYO –
RESEARCH TRIANGLE PARK, N.C., Dec. 19, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that data from the open-label extension (OLE) of the APeX-2 trial of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older have been published online by the Journal of Allergy and Clinical Immunology: In Practice (JACI: In Practice).
“As detailed in this manuscript, long-term prophylaxis with ORLADEYO has enabled patients with HAE to better manage their condition, including reducing the number of HAE attacks they experience and demonstrating clinically meaningful improvement in their quality of life. I have seen firsthand in my practice evidence that the longer patients remain on ORLADEYO, the better their outcomes are,” said William R. Lumry, M.D., clinical professor of internal medicine at the University of Texas Southwestern Medical School.
“We are pleased to share long-term efficacy and safety data from APeX-2 as published in JACI: In Practice. These data further illustrate the potential lasting outcomes that can be appreciated by patients who are treated with oral, once-daily ORLADEYO. We continue to see long-term safety and effectiveness data that reinforce ORLADEYO as an important treatment option for patients with HAE, and we look forward to sharing additional real-world evidence at upcoming medical congresses,” said Dr. Ryan Arnold, chief medical officer of BioCryst.
APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group, three-part study evaluating ORLADEYO versus placebo for the prevention of HAE attacks in 121 patients with HAE Type I or Type II. In part 3 of the study (weeks 49-96), all patients (n=81) were treated with open-label ORLADEYO at 150 mg. The primary endpoint of the OLE was long-term safety and tolerability, while secondary endpoints included HAE attack rates and quality of life (QoL).
The authors concluded that ORLADEYO was generally well tolerated, provided rapid and sustained reductions in HAE attacks and improved QoL over the study duration of 96 weeks. The results from part 3 of APeX-2 were first presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in July 2021.
Additional details can be found in the manuscript, which is available online at jaci-inpractice.org. JACI: In Practice is an official journal of the American Academy of Allergy, Asthma, and Immunology (AAAAI).
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.
U.S. Indication and Important Safety Information
INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.
IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.
The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.
A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C).
Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in pediatric patients