Orladeyo (berotralstat), an oral therapy designed to reduce or prevent hereditary angioedema (HAE) attacks, is now available in Ireland. The Irish Health Services Executive (HSE) recommended Orladeyo as a prophylactic, or preventive, treatment for HAE in adults and adolescents, ages 12 and older. Biocryst Pharmaceuticals, the company marketing…
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,…
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of donidalorsen as a preventive treatment for swelling attacks in adults and children, ages 12 and older, with hereditary angioedema (HAE). According to Ionis Pharmaceuticals, the company developing the therapy, the…
An investigational gene-editing therapy for hereditary angioedema (HAE) called P-KLKB1-101 showed it could safely work as intended in preclinical studies, according to recent data. The treatment, developed by Poseida Therapeutics, successfully edited the KLKB1 gene and lowered levels of the kallikrein enzyme across experiments in lab-grown human liver…
Initiating treatment with sebetralstat within a few minutes of the start of a hereditary angioedema (HAE) attack was associated with it resolving significantly faster than when treatment was delayed by more than two hours, according to new findings from the KONFIDENT Phase 3 trial. Sebetralstat also led to…
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…
A new mutation in the SERPING1 gene was identified as the cause of hereditary angioedema (HAE) in several members of a Han Chinese family, a study reports. Blood tests revealed varied levels of C1-INH, the protein encoded by the SERPING1 gene and used to help diagnose the condition, in affected…
Garadacimab appears to be safe for long-term use as prophylaxis, or preventive treatment, reducing the frequency of swelling attacks in adults and adolescents with hereditary angioedema (HAE), according to an interim analysis of data from an ongoing Phase 3 extension study sponsored by its developer, CSL…
The European Commission has granted orphan medicinal product designation (OMPD) to navenibart, an experimental long-acting therapy for hereditary angioedema (HAE). The designation is aimed at providing incentives for the development of therapies that are meant to diagnose, prevent, or treat rare diseases, or those affecting fewer than 10,000 people…
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