According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access to Medicine Scheme (EAMS). This occurred following a positive finding from the country’s Medicine and Healthcare Products Regulatory Agency (MHRA). The drug will be permitted for use by patients who are at least twelve years old and living with the rare disease hereditary angioedema.
About Hereditary Angioedema (HAE)
Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the SERPING1 gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attacks as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.
The drug is intended to be taken in order to prevent swelling attacks from occurring, as opposed to stopping an attack that is in progress. Orladeyo is orally available and is meant to be taken once per day, making it a more convenient option for patients that has the potential to significantly improve their overall quality of life.
About the Early Access to Medicine Scheme (EAMS)
Medicines under EAMS are generally intended to treat severe and potentially life-threatening illnesses that lack effective therapeutic options and have an urgent unmet medical need. Alternatively, these medicines may offer major advantages over current treatments. The EAMS allows for the medicine to be used by patients before it is officially granted the European Commission’s marketing authorization.
The European Medicines Agency (EMA) is in the midst of reviewing the marketing application for Orladeyo with an official verdict expected to be reached by spring of 2021.