Orladeyo Now Approved in Saudi Arabia to Prevent HAE Attacks

Developer cites 'significant need' in country for HAE treatment options

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by Steve Bryson, PhD |

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Regulatory authorities in Saudi Arabia have approved daily Orladeyo (berotralstat) to prevent hereditary angioedema (HAE) attacks in adult and adolescent patients, ages 12 and older.

“There is a significant need for new treatment options for HAE in Saudi Arabia,” Charlie Gayer, chief commercial officer of BioCryst Pharmaceuticals, the therapy’s developer, said in a company press release. “With this approval, we continue to deliver on our commitment to bringing our oral, once-daily prophylactic [preventive] therapy to as many HAE patients around the world as possible.”

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Orladeyo Shown to Prevent Attacks and Improve Quality of Life in Trial

The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, follows similar decisions in the U.S., Europe, the U.K., Japan, the United Arab Emirates (UAE), and more recently, Canada and Switzerland.

In Saudi Arabia, Orladeyo will be marketed by NewBridge Pharmaceuticals, following a supply and distribution agreement established in 2021 with BioCryst. That agreement covers Iraq and the Gulf Cooperation Council (GCC), which includes Bahrain, Oman, Kuwait, Qatar, the UAE, and Saudi Arabia.

Based in Dubai, UAE, NewBridge markets and licenses treatments approved in the U.S. and Europe that address unmet medical needs in the regions around the Middle East and North Africa, known as MENA.

“This marks the second market in the MENA region in which Orladeyo has been approved for patients living with HAE, building on the approval in the UAE last year,” said Joe Henein, NewBridge’s president and CEO.

“We are pleased with the decision from the Saudi FDA and we are committed to continuing our work with BioCryst to bring this important therapy to patients in the GCC who are in search of a more optimal treatment option to help manage their HAE,” Henein added.

Orladeyo in clinical trials

HAE is characterized by the overproduction of bradykinin, a molecule that regulates blood pressure and inflammation by stimulating blood vessels to dilate, or widen. Swelling in angioedema occurs when bradykinin is activated in the deep layers of the skin.

Given once daily as an oral capsule (150 mg), Orladeyo is designed to prevent HAE attacks by blocking plasma kallikrein, a bradykinin precursor. The preventive therapy also aims to ease the burden of standard treatment alternatives that are administered via under-the-skin (subcutaneous) or into-the-vein (intravenous) injections.

Orladeyo’s approvals were mainly supported by early data from two ongoing clinical trials sponsored by BioCryst. One, the Phase 3 APeX-2 study (NCT03485911), which enrolled 121 participants, is expected to conclude in September 2023. The other, an open-label Phase 2/3 trial called APeX-S (NCT03472040) involving 386 patients with HAE type 1 or 2, is slated to end in February 2024.

Trial data demonstrated that about a year of Orladeyo treatment effectively and sustainably reduced the monthly HAE attack rate and the need for standard-of-care treatment. Patients also reported more symptom-free days, improved quality of life, and overall treatment satisfaction than those on a placebo.

In both studies, the therapy was generally safe and well-tolerated, with gastrointestinal problems as the most common treatment-related adverse events. These side effects were usually reported shortly after treatment initiation and resolved on their own.

A recent analysis of APeX-S trial data also showed that switching from under-the-skin injections of Takhzyro (lanadelumab), a standard HAE therapy, to oral Orladeyo boosted the quality of life for trial participants.