Hereditary angioedema (HAE) is a potentially life-threatening disease, and so is Fibrodysplasia ossificans progressiva (FOP). The former affects approximately 1 in 10,000 to 1 in 50,000 people, while the former affects 1 per 735,000 to 1 per 2 million births. However, victims of HAE in the United Arab Emirates (UAE) have a reason to smile after BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) obtained a marketing authorization for ORLADEYO® (berotralstat).
The oral, once-daily treatment is reportedly a potential option for patients and physicians. One capsule per day is heaven-sent in decreasing the activity of plasma kallikrein. However, doses of more than150 mg once daily are not recommended. And whether or not it can be used during pregnancy or if it affects milk production remains unclear.
“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,” Henrik Balle Boysen, COO of HAE International, explained.
Supporting the Availability of ORLADEYO Across the Globe
HAE International is one of the many global non – profit networks dedicated to helping improve people’s lives. Nonetheless, BioCryst has also established a supply and distribution agreement with NewBridge Pharmaceuticals to assist in commercialization efforts in the UAE.
NewBridge has the necessary pharmaceutical platform of services and expertise to accelerate the availability of ORLADEYO to patients across the globe. In addition, it shares in the vision for BioCryst to bring innovative medicines to patients living with rare diseases, which are very common in the MENA region.
The Approval of ORLADEYO by UK’s National Institute for Health and Care Excellence
ORLADEYO® (berotralstat) has also found its way into the UK market. Thanks to the United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE) approval. HAE patients in England, Wales, and Northern Ireland are among the millions of UK residents who benefit from this recommendation.
From the Phase 3 APeX-2 trial findings, ORLADEYO exhibited a primary endpoint. Thanks to a significant reduction of HAE attacks vs. placebo at 24 weeks.