2 New Countries Approve Orladeyo as 1st Therapy to Prevent HAE Attacks

Regulatory health agencies in Canada and Switzerland have approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) as the first daily oral therapy to prevent hereditary angioedema (HAE) attacks.

With these approvals, Orladeyo will be available in the two countries for the routine prevention of HAE attacks in patients ages 12 and older.

“HAE carries with it a severe burden on patients and their families, much of which stems from the unpredictable attacks they experience even when actively managing their disease,” Jacquie Badiou, president of HAE Canada, said in a press release.

“I believe the first oral preventive therapy for HAE will be a welcome option for many Canadians who are living with this rare condition,” Badiou said, adding, “We have seen important treatment advancements in recent years.”

Orladeyo is expected to ease the burden of standard subcutaneous — under-the-skin — and intravenous, or into-the-vein, alternatives, which is expected to improve patients’ quality of life and independence.

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In a separate press release, Henrik Balle Boysen, HAE International’s executive vice president and chief operating officer, said that Orladeyo “offers people with HAE in Switzerland and their physicians the first orally administered non-steroidal therapy for preventing HAE attacks and provides the community with another vitally important treatment option.”

The regulatory decisions — made by Health Canada and Swissmedic — come about nine months after both agencies agreed to review BioCryst’s application seeking Orladeyo’s approval.

The new approvals follow similar rulings in the U.S., the European Union, and other countries. The U.S. approved the therapy in December 2020, with the decision in the EU following in May 2021. In Japan, the therapy was approved in January 2021.

“We appreciate Health Canada’s thorough and timely review of Orladeyo, and with this authorization we are one step closer to bringing this important treatment option to Canadians living with HAE,” said Jared Rhines, general manager of BioCryst Canada.

After regulatory approval, the Pan-Canadian Pharmaceutical Alliance starts price negotiations with the therapy’s manufacturer on behalf of public payers — including all provincial governments and the federal government. Each Canadian province and territory will then decide whether to fund the therapy.

“We look forward to working with stakeholders across the country to ensure these Canadians can receive timely and appropriate access to Orladeyo,” Rhines added.

In Switzerland — where every person is obliged to have basic health insurance — a therapy can only be reimbursed if the Federal Department of Home Affairs deems it cost-effective and adds it to the so-called “specialty list.”

“We have made significant progress in making Orladeyo available to many patients in Europe since we received European Commission approval,” said Waldemar Heiduk, CEO of BioCryst Pharma Deutschland GmbH.

“We look forward to launching Orladeyo in Switzerland soon, pending finalization of our reimbursement plans,” Heiduk said.

Health authorities in Israel also are currently reviewing the therapy for the same indication.

Given as an oral capsule in a dose of 150 mg once a day, Orladeyo is designed to prevent HAE attacks by blocking plasma kallikrein. That is a precursor of bradykinin, a pro-inflammatory molecule overly produced in HAE patients that triggers sudden swelling and pain attacks.

Orladeyo’s regulatory applications and approvals were mainly supported by early results of two ongoing, BioCryst-sponsored global clinical trials: the placebo-controlled Phase 3 APeX-2 study (NCT03485911) and the open-label Phase 2/3 APeX-S trial (NCT03472040).

Both trials are assessing whether the therapy can safely and effectively prevent swelling attacks in adults and adolescents, ages 12 and older, with HAE types 1 and 2. APeX-2 trial enrolled 121 patients, while APeX-S recruited 386 patients.

Results from these studies showed that about a year of treatment with Orladeyo’s approved dose effectively and sustainably lowered the monthly attack rate and the need for standard-of-care treatment.

Patients on this dose also reported overall treatment satisfaction, meaningful improvements in quality of life, and more symptom-free days, compared with those on a placebo.

Orladeyo has been generally safe and well-tolerated in both studies. The most common adverse events relative to the placebo were gastrointestinal problems, which were typically reported shortly after treatment initiation and generally resolved on their own.

APeX-2 is expected to conclude in September 2023 and APeX-S to end in February 2024.

“I believe this oral, once-daily prophylactic [preventive] therapy will be a meaningful treatment option for patients who need a prophylactic therapy to help manage their HAE by reducing the frequency of attacks,” said Stephen Betschel, MD, chair of the Canadian Hereditary Angioedema Network.

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