Canadian agency recommends cost of Orladeyo be covered

CADTH also seeks price reduction from current annual cost of $310K per patient

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by Andrea Lobo |

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The Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended Orladeyo (berotralstat) be reimbursed by public drug plans when used for the routine prevention of hereditary angioedema (HAE) attacks in patients 12 years and older.

“We are still in need of additional options to help people living with HAE better manage their disease, despite the incredible progress we have made to date in addressing the treatment burden,” Daphne Dumbrille, chief operations officer at HAE Canada, said in a press release.

“[This] announcement is a welcome one for the HAE community in Canada, as it should help increase access to this oral prophylactic treatment across our country,” Dumbrille added.

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Orladeyo estimated to cost public plans about $310,463 per patient annually

The agency recommends a price reduction, since “Orladeyo does not represent good value to the health care system at the public list price.” Based on public list prices, Orladeyo is estimated to cost public plans around $310,463 per patient annually, corresponding to approximately $93 million over the next three years.

CADTH also recommends that Orladeyo be reimbursed following the same criteria as for Takhzyro (lanadelumab), another approved therapy to prevent HAE attacks. In addition, it states that Orladeyo should not be used in combination with other medications for the long-term prevention of HAE attacks.

“In issuing this positive recommendation, CADTH is opening the door to many Canadian HAE patients being able to access Orladeyo,” said Stephen Betschel, MD, chair of the Canadian Hereditary Angioedema Network.

HAE is characterized by sudden swelling attacks in the deeper layers of the skin driven by the overproduction of a signaling molecule called bradykinin, which is controlled by the protein kallikrein.

Orladeyo is an oral medication developed by BioCryst Pharmaceuticals to prevent HAE swelling attacks. It works by blocking the activity of kallikrein and preventing bradykinin levels from getting too high, thereby preventing swelling attacks.

Health Canada approved Orladeyo in June 2022 for the routine prevention of swelling attacks in HAE patients 12 years and older.

At the time, the decision was supported mainly by early data from two clinical trials: a placebo-controlled Phase 3 study called APeX-2 (NCT03485911) and a Phase 2/3 open-label extension study called APeX-S (NCT03472040).

Results from both studies showed that about a year of treatment with Orladeyo reduced the number of monthly HAE attacks and the need for standard-of-care treatment.

In issuing this positive recommendation, CADTH is opening the door to many Canadian HAE patients being able to access Orladeyo.

Orladeyo found to reduce frequency of HAE attacks in clinical trials

CADTH’s recommendation for reimbursement is based on data from APeX-2 and another Phase 3 trial called APeX-J (NCT03873116). Both trials enrolled HAE patients who had experienced at least two attacks in the 56 days before the studies started.

Compared with a placebo, daily treatment with 150 mg of Orladeyo for 24 weeks (around six months) significantly reduced the frequency of HAE attacks. In APeX-2, this reduction was 44.2% and in APeX-J, it was 49.1%.

Moreover, patients on Orladeyo in APeX-2 reported approximately 13 fewer symptoms than those in the placebo group.

“BioCryst is delighted that Orladeyo has received this positive recommendation from CADTH,” said Anand Janack, vice president and general manager at BioCryst Canada. “I am grateful to our team here at BioCryst and our many partners in the HAE community who underscored the value of ensuring Canadian patients have access to an oral, once-daily therapy for this rare, debilitating disease.”