Kalvista Pharmaceuticals has submitted an application asking Japan’s Ministry of Health, Labour and Welfare (MHLW) to approve its oral therapy sebetralstat as an on-demand treatment to manage swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. “The submission of our [new drug application] for sebetralstat…
Intellia Therapeutics is planning to submit an application requesting the approval of NTLA-2002, its gene-editing therapy for hereditary angioedema (HAE), to U.S. regulators in late 2026. The company recently announced it would be discontinuing programs for other diseases, as well as reducing its workforce, to focus efforts…
A European Medicines Agency (EMA) committee has recommended that the antibody-based therapy garadacimab be approved as a once-monthly preventive treatment to reduce the risk of swelling attacks in people with hereditary angioedema (HAE) ages 12 and older. The positive opinion from the EMA’s Committee for Medicinal Products…
There are notable differences between hereditary angioedema with normal C1-inhibitor function (HAE nC1-INH) and idiopathic angioedema of unknown etiology (AE-UNK), even though they share many features, a real-world Canadian study indicates. Some of the differences include the time to reach a diagnosis, attack triggers and characteristics, and treatment…
Theratechnologies said it will ask Health Canada next year to approve donidalorsen, an experimental injection therapy designed to reduce the risk of swelling attacks in people with hereditary angioedema (HAE). The company agreed to acquire exclusive rights to commercialize the treatment in the country. Donidalorsen, originally developed…
Astria Therapeutics is planning the launch of a Phase 3 clinical trial in early 2025 to test different dosing schedules of navenibart, its preventive therapy for hereditary angioedema (HAE). The goal, according to Astria, is to provide flexibility for patients, with different dosing options, should the treatment candidate…
DRI Healthcare Trust, an investment firm, has acquired rights to royalty interest in sebetralstat, an on-demand treatment for hereditary angioedema (HAE) that’s being considered for approval in several countries, including the U.S. For Kalvista Pharmaceuticals, the therapy’s developer, the deal could be worth more than $175 million,…
Most people with hereditary angioedema (HAE) who received treatment with a 50 mg dose of the gene-editing therapy NTLA-2002 in a Phase 2 clinical trial were free from swelling attacks over nearly four months, according to a new study. Compared with a placebo, the 50 mg dose led…
The case of an elderly man who developed acquired angioedema associated with an underlying blood disorder called monoclonal gammopathy was described in a new report, with the researchers noting the condition is “very rare but serious.” The man’s angioedema was controlled only after the underlying monoclonal gammopathy was successfully…
An algorithm that takes into account factors such as the frequency and location of swelling attacks may help distinguish chronic histaminergic angioedema (CHA), which is a form of angioedema, from chronic spontaneous urticaria (CSU), a disorder with similar symptoms. That’s according to a study, “Predictive…
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