Argentina approves oral Orladeyo to prevent HAE attacks

In clinical trials, therapy was found to reduce number of HAE swelling attacks

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Argentina’s regulatory body has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) to prevent swelling attacks in adults and adolescents, ages 12 and older, who have hereditary angioedema (HAE).

The decision by the National Administration of Drugs, Foods, and Medical Devices (ANMAT) comes a few months after the approval of Orladeyo in Chile, its first in the Latin American region.

Under a 2022 agreement with BioCryst, Pint Pharma is responsible for securing all regulatory approvals and for marketing the oral prophylactic (preventive) therapy across Latin America, including in Chile and Argentina.

“We continue to make strides to bring Orladeyo to patients living with HAE in Latin America in collaboration with our partner, Pint Pharma,” Charlie Gayer, chief commercial officer of BioCryst, said in a company press release. “We applaud ANMAT’s decision to grant approval to our oral, once-daily prophylactic therapy for HAE, which paves the way for patients living with HAE to receive Orladeyo to help improve management of their HAE attacks.”

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Orladeyo is a hard gelatin capsule given once daily

In HAE, a protein called kallikrein becomes overactive, leading to the overproduction of a signaling molecule called bradykinin. Too much bradykinin makes blood vessels widen and causes fluid to leak out into the surrounding tissue, resulting in swelling attacks.

Orladeyo is a once-daily hard gelatin capsule containing berotralstat, a molecule that works by inhibiting the activity of kallikrein, thereby preventing bradykinin levels from getting too high and reducing the number of swelling attacks.

Orladeyo was first approved by the U.S. Food and Drug Administration in late 2020 to prevent swelling attacks in adults and adolescents with HAE, ages 12 and older, based on evidence from a Phase 3 clinical trial called APeX-2 (NCT03485911).

The study included 120 patients who had at least two swelling attacks during a period of about two months before starting the trial. Patients assigned to receive a placebo had an average of 2.4 swelling attacks per month, compared with 1.3 in those taking 150 mg of Orladeyo.

In a long-term, open-label Phase 2/3 clinical study called APeX-S (NCT03472040), patients completing 96 weeks (nearly two years) of treatment with 150 mg of Orladeyo had a mean of 0.8 swelling attacks per month, with a median of zero for most of the study duration.

Orladeyo also won approval in Europe, Japan, Israel, and Saudi Arabia.