Orladeyo approved in Brazil for HAE patients 12 and older

1st oral preventive medication approved for HAE swelling attacks in Brazil

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The once-daily oral therapy Orladeyo (berotralstat) has been approved in Brazil to prevent swelling attacks in people, 12 and older, with hereditary angioedema (HAE).

The approval by the Brazilian Health Regulatory Agency (ANVISA) makes Orladeyo the first oral prophylactic (preventive) therapy authorized for HAE patients in the country.

“The approval of Orladeyo by ANVISA is excellent news for the hereditary angioedema patient community in Brazil,” Valnei Canutti, chief scientific officer at Pint Pharma, said in a press release.

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Orladeyo’s approval in Brazil follows similar approvals in Argentina, Chile

Pint, based in Austria, is responsible for obtaining approval of Orladeyo and commercializing it in Latin America under a 2022 agreement struck with the therapy’s original developer, BioCryst Pharmaceuticals. Orladeyo’s approval in Brazil came a few months after the therapy received similar approvals in Argentina and Chile last year.

“In addition to providing direct benefits to patients, the availability of Orladeyo in Brazil highlights our continued commitment to advancing research and innovation in the field of rare disease healthcare,” said David Munoz, CEO of Pint Pharma.

Fernanda Bertasi, general manager of Pint Pharma in Brazil, added the approval “is a great achievement for patients and a proof of the successful collaboration between the medical community, regulators, and pharmaceutical companies in Brazil to enhance the treatment options.”

HAE is caused by mutations that result in excessively high levels of a signaling molecule called bradykinin. High bradykinin levels cause blood vessels to leak fluid, which results in the swelling attacks that characterize the disorder.

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Orladeyo works to lower bradykinin levels to reduce risk of swelling attacks

The active agent in Orladeyo works to block the activity of an enzyme, called kallikrein, that helps produce bradykinin. By inhibiting this enzyme, Orladeyo works to lower bradykinin levels and ultimately reduce the risk of swelling attacks.

“This innovative oral therapy represents a breakthrough for those living with this rare condition, offering an effective and convenient long-term prophylaxis option, preventing hereditary angioedema (HAE) attacks,” Canutti said.

Orladeyo has been approved in the U.S. since 2020 for HAE patients 12 and older. The therapy has also received similar approvals in Europe, Japan, Israel, and Saudi Arabia.

The approvals were mainly based on data from the Phase 3 APeX-2 trial (NCT03485911), which compared Orladeyo against a placebo in 120 HAE patients. It showed participants given the therapy had lower rates of swelling attacks.

Long-term data from an extension study, called APeX-S (NCT03472040), showed the majority of participants who took Orladeyo for two years had no swelling attacks for most of this time.