Orladeyo Approved in United Arab Emirates to Prevent HAE Attacks

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by Marta Figueiredo PhD |

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The United Arab Emirates (UAE) has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) for preventing hereditary angioedema (HAE) attacks in patients, 12 and older.

“As the first targeted oral, once-daily treatment, Orladeyo provides an important new treatment option for patients and physicians,” Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global nonprofit that supports local HAE patient groups and organizations, said in a press release.

“While there is still more work to be done to raise awareness to support earlier diagnosis and treatment, the approval of Orladeyo is an important advancement for HAE patients in the UAE,” he added.

The approval by the country’s ministry of health and prevention follows similar decisions in the U.S., the European Union, the U.K., and Japan. The therapy is also currently being reviewed by health authorities in Israel, Canada, and Switzerland for the same indication.

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Orladeyo will be marketed in the UAE by NewBridge Pharmaceuticals following a supply and distribution agreement with BioCryst that covers also Saudi Arabia, Qatar, Bahrain, Oman, Kuwait, and Iraq.

Headquartered in Dubai, NewBridge licenses and markets approved therapies in the U.S. and Europe that address unmet medical needs in the Middle East and North Africa (MENA) regions.

“With many prevalent rare diseases in the MENA region, I am personally inspired, and we at NewBridge are proud, to be part of this partnership with BioCryst for the UAE and a number of other markets,” said Joe Henein, NewBridge’s president and CEO.

“This partnership supports our mission by providing access to an important new therapy for HAE patients in a hope that we can help ease their suffering and support them to live better lives,” Henein added.

Charlie Gayer, BioCryst’s chief commercial officer, said that “NewBridge is the right partner for BioCryst as they share our vision to bring innovative medicines to patients living with rare diseases.”

“With experience across regulatory, medical and commercial, and strong local relationships with key stakeholders, NewBridge will help accelerate our efforts to bring Orladeyo to patients across the globe by providing a much-needed new option to HAE patients in the UAE,” Gayer added.

Orladeyo is designed to prevent the levels of bradykinin, an inflammatory molecule overly produced in HAE patients, from rising too high and triggering sudden swelling and pain attacks.

Given as an oral capsule (150 mg) once a day, the preventive therapy is expected to also ease the burden of standard under-the-skin and into-the-vein alternatives, improving patients’ quality of life and independence.

Regulatory applications and approvals of Orladeyo were mainly supported by positive data from two ongoing clinical trials: the global, placebo-controlled Phase 3 APeX-2 study (NCT03485911) and the open-label Phase 2/3 APeX-S trial (NCT03472040).

Both are investigating the therapy’s safety and effectiveness at preventing swelling attacks in people, 12 and older, with HAE types 1 or 2. APeX-2, involving 121 patients, is expected to conclude in September 2023, while APeX-S is following 386 participants until February 2024.

APeX-2’s results showed that, compared with a placebo, a daily dose of 150 mg of Orladeyo significantly reduced the number of monthly HAE attacks by 44% during the first six months of treatment, and that this rate remained low through one year. The number of attacks dropped from a mean of 2.9 per month at the study’s start to one per month at nearly one year.

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Similarly, patients treated with Orladeyo’s 150 mg dose for about one year in the APeX-S trial had a mean of 0.8 HAE attacks per month.

Moreover, Orladeyo-treated patients reported meaningful improvements in quality of life and overall treatment satisfaction. The therapy was also associated with a significantly lower need for standard-of-care medications and with more symptom-free days relative to a placebo.

In both studies, Orladeyo has been reported to be generally safe and well-tolerated.