The European Commission (EC) has granted orphan drug designation to NTLA-2002, an investigational gene-editing therapy that Intellia Therapeutics is developing for hereditary angioedema (HAE). The designation is given to therapies with the potential to improve medical care for people with serious conditions that affect up to five of…
News
Donidalorsen, an experimental treatment in the pipeline of Ionis Pharmaceuticals, continues to prevent swelling attacks when taken for up to two years, allowing a better quality of life for adults with hereditary angioedema (HAE). These results come from an ongoing open-label extension study (NCT04307381) in HAE…
Most older adults with hereditary angioedema (HAE) have more frequent and severe swelling attacks, and more chronic diseases that combine to have a negative impact on their quality of life, results from a focus group study show. Other age-related difficulties included financial barriers to accessing medication. Despite these challenges,…
Pharvaris is now gearing up startup activities with clinical site investigators and staff to launch a Phase 3 trial next year of PHVS416 — an immediate-release capsule formulation of deucrictibant — as an on-demand treatment for hereditary angioedema (HAE). The Switzerland-based clinical-stage company recently met with the U.S. Food…
A 58-year-old woman developed angioedema of the small bowel after three years of taking lisinopril, an angiotensin-converting enzyme (ACE) inhibitor often used for high blood pressure. The woman switched to amlodipine, a different blood pressure-lowering medication, and her signs and symptoms of angioedema resolved rapidly. Her case was…
Treatment with rituximab — approved for certain cancers to reduce the levels of antibody-producing immune cells — is effective to control swelling attacks in people with acquired angioedema, particularly in cases where the disease is linked to an underlying lymphoma, or a blood cancer that develops in the lymphatic…
Even with long-term preventive treatment, many people with hereditary angioedema still sometimes have swelling attacks that require on-demand treatment, a review study finds.
Children with hereditary angioedema (HAE) may have dozens of swelling attacks until they receive a correct diagnosis of the disease, according to a study in a hospital in Turkey, where the median delay was three years. “Awareness of the symptoms of HAE is necessary and correct diagnosis is…
A 3-point score increase is the minimum change on the widely used Angioedema Control Test (AECT) to reflect meaningful improvement in disease control for people with recurrent angioedema, a study showed. The AECT was developed to help patients keep score of disease control over a recall period, but…
The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to Intellia Therapeutics’ NTLA-2002, an experimental gene-editing therapy to prevent swelling attacks in people with hereditary angioedema (HAE). A PRIME designation supports the development of experimental therapies that address unmet medical needs. Eligibility for this status is…
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