NTLA-2002, the investigational gene-editing therapy from Intellia Therapeutics, reduced the number of monthly swelling attacks by a mean of 98% among people with hereditary angioedema (HAE) who were treated in the Phase 1 portion of an ongoing Phase 1/2 clinical trial. The one-time treatment also led to sustained,…
News
Treatment with donidalorsen to prevent swelling attacks led to fewer of them and gains in quality of life for adults and teens with hereditary angioedema (HAE), according to final data from a Phase 3 trial and interim findings from its open-label extension study. The patients who switched from other prophylactic…
The risk of venous thromboembolism (VTE), which occurs when a blood clot forms in a vein and obstructs blood flow, is 2.5 times higher in people with hereditary angioedema (HAE) than in their relatives without it, a study of Swedish families shows. The risk for VTE was higher in…
Deucrictibant, an experimental treatment for hereditary angioedema (HAE), now has shown promising efficacy as an on-demand and prophylactic, or preventive, treatment in three patients with acquired angioedema. The findings, from a small clinical trial, suggest that “deucrictibant has the potential to effectively and safely treat and prevent…
Genetic variants in the CC2D2B gene may affect disease severity in hereditary angioedema (HAE) types 1 and 2, according to a new study. “We demonstrated, on a large cohort of [people with HAE types 1 and 2], the importance of the CC2D2B gene as a disease-modifying factor,” the researchers…
Orladeyo (berotralstat), an oral therapy designed to prevent swelling attacks in adults and adolescents with hereditary angioedema (HAE), has now been approved in Mexico. The approval by the Mexican Federal Commission for Protection against Health Risks (COFEPRIS) covers the use of Orladeyo as a prophylactic, or preventive, treatment for HAE…
Treatment with oral Orladeyo (berotralstat) led to significantly fewer disease-related or all-cause hospitalizations and emergency room visits by people with hereditary angioedema (HAE) in the U.S., according to a real-world claims analysis. “The data presented here represent the first documentation that prescribing Orladeyo significantly reduces healthcare resource utilization,”…
Hereditary Angioedema (HAE) Awareness Day is observed every May 16 to call attention to a disorder that affects an estimated 1 in 50,000 to 150,000 people globally. Called “hae day :),” the observance targets targets policymakers, industry representatives, public authorities, researchers, and healthcare professionals, along with the general public.
The unusual case of a woman with drug-induced angioedema that led to a rare complication called acute compartment syndrome (ACS) in all four of her limbs was described in a recent report from Japan. ACS is a disorder in which the pressure within one of the body’s compartments —…
KalVista Pharmaceuticals plans to submit an application to the U.S. Food and Drug Administration (FDA) in June seeking approval of its oral therapy sebetralstat for the on-demand treatment of hereditary angioedema (HAE), the company announced this week while unveiling its strategic plan for fiscal year 2025. Similar applications…
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