Orladeyo Approval for HAE Under Review in Canada, Switzerland

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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Regulatory health agencies in Canada and Switzerland are reviewing BioCryst Pharmaceuticals’ applications for the approval of Orladeyo (berotralstat) in their countries for preventing hereditary angioedema (HAE) attacks in patients ages 12 and older.

The applications — to Heath Canada and Swissmedic — followed Orladeyo’s recent approvals in the U.S., the European Union, and other countries as the first oral treatment to prevent HAE attacks.

We are making significant progress with our goal to bring Orladeyo to HAE patients around the world,” Jon Stonehouse, BioCryst’s president and CEO, said in a press release.

The review process for new therapies usually takes less than one year with Health Canada and about 1.5 years with Swissmedic.

“Currently there are no targeted oral HAE therapies available in Canada or Switzerland for patients living with HAE,” Stonehouse said, adding that “if approved, Orladeyo has the potential to be an important new treatment option for Canadian and Swiss HAE patients and physicians.”

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The announcement comes two months after a similar application was filed with regulatory authorities in Israel. To date this year, Orladeyo also has been granted approval in Japan and the U.K.

An oral capsule taken once daily, Orladeyo works by blocking plasma kallikrein, a precursor of an inflammatory molecule called bradykinin that’s overly produced in HAE patients. When present at excess levels, bradykinin causes blood vessels to widen and become leaky, leading to sudden swelling and pain attacks.

By preventing such high bradykinin levels, Orladeyo is designed to reduce HAE attacks. It’s also been touted for its easier mode of administration compared with standard injectable treatments. Oral therapies have been found to improve patients’ quality of life and level of independence.

Orladeyo’s regulatory applications were supported by the early results of two ongoing clinical trials involving patients with HAE type 1 and type 2. The placebo-controlled Phase 3 APeX-2 trial (NCT03485911) included 121 patients while the open-label Phase 2/3 APeX-S trial (NCT03472040) had 386 participants. Both APeX-2 and APeX-S are sponsored by BioCryst.

The two trials are evaluating the therapy’s safety and effectiveness at preventing attacks in adults and adolescents, ages 12 and older. APeX-2 is slated to conclude in September 2023 and APeX-S will run until February 2024.

Data from these studies showed that about a year of treatment with Orladeyo effectively and sustainably reduced the rate of monthly HAE attacks and the need for standard-of-care treatment. It also was found to improve patients’ quality of life.

To date, the therapy has been generally safe and well-tolerated in both studies, with the most common adverse events being gastrointestinal problems. Such side effects generally occurred shortly after patients started on Orladeyo and became less frequent over time, typically resolving on their own.