News

Bradykinin, a key molecular player involved in swelling attacks in hereditary angioedema patients, does so by signaling through the cyclooxygenase (COX) proteins, which may be targeted with common anti-inflammatory medications such as ibuprofen and diclofenac, a study has found. By uncovering additional molecules participating in…

Intellia Therapeutics announced the first preclinical results supporting a gene therapy candidate for hereditary angioedema (HAE). In animal models, the product markedly and sustainably lowers kallikrein activity to levels of expected therapeutic benefit, anticipated to lessen angioedema attacks in patients. The early data comes on the…

Haegarda, a C1-INH replacement medication by CSL Behring, is now available in the Canadian province of Quebec for the preventive treatment of swelling attacks in adults and adolescents with hereditary angioedema (HAE), the company announced. “The availability of a new treatment option to help health care professionals…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to provide…

In Italy, people with hereditary angioedema (HAE) types 1 or 2 are not at an increased risk of premature death relative to the general population, and swelling that blocks the airway is not the main cause of death, a new study has found. The study, “Life…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

BioCryst Pharmaceuticals has submitted an application to the Japanese regulatory authority asking that oral berotralstat (BCX7353) be approved for the prevention of swelling attacks in people with hereditary angioedema (HAE). The new drug application (NDA), submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, follows…

The European Medicines Agency (EMA) has approved a type II variation for a new facility to produce Ruconest (conestat alfa), Pharming’s lead therapy for acute attacks in hereditary angioedema (HAE). The new facility is expected to be fully operational by 2021 and to increase the production…

A new diagnostic tool has been identified for people with hereditary angiodema (HAE) and normal C1 inhibitor activity, potentially establishing the first method that doctors can use to properly diagnose this genetic condition, a study suggests. The study, “sgp120 and the contact system in hereditary…

Researchers have developed a new tool that measures disease activity specifically in people with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE), which could prove useful in the medical care of patients and for assessing outcomes in clinical trials. The Hereditary Angioedema Activity Score (HAE-AS) was described in the…