News

UK innovation passport issued to sebetralstat as treatment for HAE

Sebetralstat, an oral small molecule being developed by KalVista Pharmaceuticals as an on-demand treatment for hereditary angioedema (HAE), has been issued an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). An innovation passport is the first step into the U.K.’s Innovative Licensing and Access…

Sebetralstat found effective in trial as on-demand treatment for HAE

On-demand treatment with the experimental oral medication sebetralstat was effective for resolving swelling attacks in people with hereditary angioedema (HAE) — meeting all endpoints, or goals, in the Phase 3 KONFIDENT trial — top-line data show. Based on the positive findings, sebetralstat’s developer KalVista Pharmaceuticals is planning…

Vision loss caused by optic nerve swelling in man with HAE: Report

A 60-year-old man with hereditary angioedema (HAE) caused by an unknown mutation developed visual loss due to swelling of the optic nerve in both eyes, according to a case reported in Italy. His symptoms eased after starting treatment with Berinert (human C1 esterase inhibitor), an intravenous (into-the-vein) C1…

Single dose of NTLA-2002 seen to reduce monthly HAE attacks 95%

Intellia Therapeutics’ experimental gene-editing therapy NTLA-2002 reduced the number of monthly swelling attacks by 95% among 10 people with hereditary angioedema (HAE) treated in the Phase 1 portion of a Phase 1/2 clinical trial, according to newly published data. The single-dose treatment also led to sustained, dose-dependent reductions in…

FDA lifts hold on deucrictibant as preventive treatment for HAE

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on deucrictibant as a prophylactic, or preventive, treatment for hereditary angioedema (HAE) attacks. Deucrictibant developer Pharvaris said it will resume clinical testing of the therapy in HAE, including restarting the open-label extension part of the…