At the 2021 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), BioCryst Pharmaceuticals, Inc. presents new long-term efficacy and safety data from studies evaluating oral, once-daily Orladeyo (berotralstat) for the prophylactic treatment of HAE.

“We continue to see improvement in key indicators that demonstrate the value of oral, once-daily Orladeyo as an excellent option for patients who want alternatives to injectable prophylactic therapies, which carry a high treatment burden,” says Dr. William Sheridan, Chief Medical Officer of BioCryst. “The data further support Orladeyo as a transformative therapy for HAE patients, including those who are already well-controlled on other therapies.”

“The data from APeX-S showed that HAE patients who switched to Orladeyo from an injectable prophylactic therapy had more than 80 percent attack free months,” says Marc Riedl, M.D., Clinical Director of the US HAEA Angioedema Center at the University of California San Diego. “These data show that, regardless of which therapy patients switched from, or when they switched, Orladeyo provided consistently low attack rates when used as a monotherapy. These important findings add to real-world evidence that this oral, once-daily therapy is a beneficial treatment option for many HAE patients.”

“Increased satisfaction and improvement in quality of life continue to be major driving factors for patients who decide to switch to an oral prophylactic option, as shown in our analysis of patients who switched from injectable prophylaxis to Orladeyo in APeX-2,” says Jonathan Bernstein, M.D., Professor of Medicine, department of internal medicine, division of allergy & immunology at the University of Cincinnati and partner of the Bernstein Allergy Group and Bernstein Clinical Research Center. “These results underscore what I see every day in clinical practice, that HAE patients want a more convenient treatment option to control their HAE attacks and reduce their overall burden of therapy.”

BioCryst ACAAI 2021 Presentation Highlights

Consistently Low HAE Attack Rates Observed in US Patients Treated with Berotralstat:

  • Highlights safety, effectiveness, and patient-reported outcomes of ORLADEYO 150 mg in U.S. patients from APeX-S who completed 12 months of treatment (n=71).
  • Following initiation of ORLADEYO, patients experienced low HAE attack rates that were sustained throughout the treatment period (median attack rate of 0.0 attacks per month through month 12), consistent with previously reported data.
  • Orladeyo was associated with prompt, sustained, statistically significant, and clinically meaningful improvements in quality of life (-10.8 change from baseline at month 1 and -13.6 change from baseline at months 6 and 12; p<0.001) as measured by the AE-QoL (angioedema quality of life questionnaire) total score. These improvements exceeded the minimal clinically important difference (-6 change).
  • Orladeyo was generally well tolerated, with no drug-related serious adverse events reported.

Berotralstat Demonstrates Low HAE Attack Rates in Patients Switching from Injectable Prophylaxis:

  • Patients at U.S. sites in APeX-S who switched to Orladeyo (n=34) had more than 80 percent attack free months for up to 12 months after switching from long-term prophylactic treatment (LTP) (lanadelumab and C1 inhibitors).
  • After switching from prior injectable prophylaxis, mean monthly attack rates were consistently low (< 0.5 attacks per month) and median attack rates remained at 0.0 attacks per month throughout 12 months of treatment with Orladeyo monotherapy.
  • The transition from injectable LTP to Orladeyo was generally well tolerated with no additional safety signals.

Improved Patient Satisfaction with Berotralstat in Patients Switching from Injectable HAE Prophylactic Treatments:

  • Treatment satisfaction in patients who switched to Orladeyo monotherapy from prior injectable LTP at U.S. sites in APeX-S was reported as assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM).
  • Statistically significant improvements were observed in convenience and global satisfaction scores in patients who switched from lanadelumab or a subcutaneous C1 inhibitor to Orladeyo (n=34), consistent with a positive experience using an oral HAE prophylactic therapy.
  • For all patients who switched to Orladeyo, the most significant improvement was observed in convenience, with scores of more than 90 points at month 12 (33.4 point improvement; p<0.001).
  • From baseline to month 12, effectiveness scores remained consistently high, supporting patients’ perception that Orladeyo is as effective as their prior prophylactic therapy.

Sustained Reduction in HAE Attack Rates Following Switch to Berotralstat: Subgroup Analysis from APeX-2:

  • Long-term efficacy and safety data were analyzed in patients who switched to Orladeyo 150 mg for parts 2 and 3 of the APeX-2 pivotal clinical trial after receiving placebo in part 1 of the trial (n=17).
  • A rapid reduction in attack rates was observed following the switch to Orladeyo, with median attack rates of 0.0 attacks per month at more than 75 percent of all timepoints.
  • Sustained reductions in mean and median HAE attack rates were observed after patients switched to Orladeyo
  • Orladeyo was generally well tolerated in patients who switched from placebo; safety data in this switch subset were consistent with those previously reported for the overall study patient population.

(Source: BioCryst)