Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration has approved Orladeyo (berotralstat) as an oral treatment to prevent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. This approval makes Orladeyo, developed by BioCryst Pharmaceuticals, the first and only oral treatment available for these patients, for whom…

The Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) attacks has completed enrollment, the company announced. Results from the trial are expected by year’s end. Meanwhile, the company has submitted a pediatric investigational plan for KVD900 to the European Medicines…

Costs are unsustainable for preventive treatment of hereditary angioedema (HAE) attacks with approved therapies Takhzyro or Haegarda, according to an analysis of six-month real-world data performed by the pharmacy benefit manager Prime Therapeutics. In addition, on-demand therapy plus Takhzyro cost $26,000 more than the same regimen plus Haegarda.

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The U.S. Food and Drug Administration (FDA) agreed to review BioCryst Pharmaceuticals‘ application requesting approval of oral berotralstat as a once-daily treatment to prevent swelling attacks in people with hereditary angioedema (HAE). ByoCryst submitted the new drug application (NDA) to the FDA in December 2019, and the agency…

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…

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…

Results of the Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) attacks will be announced by August. The company also announced it has selected KVD824 as an oral preventive therapy for HAE. A Phase 2…

Treatment with Takhzyro (lanadelumab) effectively reduces the number of attacks in patients with hereditary angioedema even in early phases of treatment, new analysis of data from the Phase 3 HELP study shows. Most recent trial results were shared in a poster, “Lanadelumab demonstrates rapid and sustained prevention of hereditary…