BioCryst Pharmaceuticals, Inc. announces new real-world data demonstrating rapid, sustained reduction of patient-reported HAE attacks and consistently low attack rates among patients 12 years and older who started on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of HAE, including patients who switched from other prophylactic therapies.

The data are being presented at the 2022 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI).

“These data are particularly exciting because consistent with our long-term clinical program data, they show that patients on ORLADEYO sustain, and even improve, attack control the longer they are on therapy,” says Dr. Ryan Arnold, Chief Medical Officer of BioCryst: “It is notable that this real-world evidence suggests that people living with HAE can maintain or improve control of their disease on ORLADEYO, regardless of prior treatment history.”

“Every HAE patient has a unique experience with their therapy, and these data demonstrate that ORLADEYO can be a very effective treatment option for patients regardless of their reported prior attack rates or prophylactic therapy history. The sustained, long-term attack rate reductions we are seeing illustrate the durability of this efficacy for patients who are looking to improve control over their HAE by switching to a therapy with a less burdensome route of administration than subcutaneous or intravenous prophylactic therapies,” says William R. Lumry, M.D., Clinical Professor of internal medicine at the University of Texas Southwestern Medical School.

The presentations at ACAAI are based on analyses from patient-reported results collected in the real-world clinical setting from BioCryst’s sole-source pharmacy, including HAE Type I and Type II patients in the United States who actively received ORLADEYO between 16 December 2020, and 20 May 2022.

BioCryst ACAAI 2022 Presentation Highlights

Consistently Low Hereditary Angioedema Attack Rates Observed with Berotralstat Regardless of Previous Prophylaxis: Real-World Outcomes:

  • This analysis assessed patient-reported HAE attack rates of patients on ORLADEYO 110 mg or 150 mg who were previously on another prophylactic therapy (n=129), including lanadelumab (n=53), a subcutaneous (SC) C1 esterase inhibitor (n=31), danazol (n=15) and an intravenous (IV) C1 esterase inhibitor (n=21). Nine patients were on a combination of prophylactic therapies.
  • Regardless of prior prophylaxis, a rapid reduction in median attack rates was observed early (1.67 attacks/month at baseline to a median attack rate of 0.33 attacks/month in days 1-90). The reduction of median attack rates was sustained throughout the 360-day treatment period.
  • Upon initiating ORLADEYO treatment, the reduction in median attack rates from baseline over the 1–360 days period was consistent for patients regardless of their prior prophylaxis therapy. The reductions after starting ORLADEYO for each prior prophylactic therapy were:
    • 77 percent reduction for patients previously on lanadelumab;
    • 64 percent reduction for patients previously on SC C1-INH;
    • 70 percent reduction for patients previously on danazol; and
    • 72 percent reduction previously on IV C1-INH.
  • The incidence of AEs reported was lower than the incidence reported in clinical trials.

Rapid and Sustained Reductions in Hereditary Angioedema Attack Rates with Long-term Berotralstat: Real-World Outcomes (Distinguished Industry Oral Presentation):

  • This analysis assessed the efficacy of ORLADEYO 110 mg or 150 mg in patients (n=128) for a treatment period of more than 270 days.
  • Patients reported a meaningful reduction in HAE attack rates when treated with ORLADEYO. An 80 percent average reduction from median baseline attack rate (1.67 median attacks/month at baseline vs. 0.33 median attacks/month during days 1-90) was observed during the initial 90-day treatment period. Regardless of baseline attack rate, patients reported a reduction in attack rates when treated with ORLADEYO.
  • The incidence of adverse events (AEs) reported was consistent with the incidence reported in clinical trials.

(Source: BioCryst)