KalVista Pharmaceuticals has started a clinical trial to test the oral medication sebetralstat as an on-demand treatment for swelling attacks in children with hereditary angioedema (HAE), ages 2-11. The launch of KONFIDENT-KID (NCT06467084) came just a few weeks after KalVista asked the U.S. Food and…
Switching to Orladeyo (berotralstat) may help reduce the frequency and duration of swelling attacks in people with hereditary angioedema (HAE) who have normal levels and activity of a protein called C1-inhibitor, according to a European case series. These data add “clinically relevant evidence” to a growing body of…
Otsuka Pharmaceutical has obtained exclusive rights to commercialize donidalorsen for hereditary angioedema (HAE) in the Asia-Pacific region. The deal is an extension of a previous license agreement that granted Otsuka the rights to commercialize the therapy in Europe. Ionis Pharmaceuticals, the therapy’s original developer, retains…
A research collaboration between Pentavere Research Group and Takeda Canada has led to the creation of one of the largest cohort datasets of patients with an ultra-rare form of hereditary angioedema (HAE) where C1 inhibitor (C1-INH) protein levels are normal. The public-private partnership seeks to “revolutionize” diagnosis and care…
KalVista Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) requesting the approval of sebetralstat, an oral on-demand treatment for people, 12 and older, with hereditary angioedema (HAE). The agency now has 60 days to determine whether the application, submitted in the form of…
An immediate-release capsule of deucrictibant that’s being developed by Pharvaris continues to show promise as an on-demand and preventive treatment for attacks of hereditary angioedema (HAE). “We believe deucrictibant is the only oral molecule with the potential to address both the on-demand and prophylactic treatment modalities for people…
Most hereditary angioedema (HAE) patients are able to take on-demand oral tablets of sebetralstat within 10 minutes of a swelling attack starting, and begin experiencing symptom relief within two hours, with complete resolution within about a day. That’s according to interim findings from an open-label extension Phase 3…
A young woman who developed angioedema of the small bowel after taking two different angiotensin-converting enzyme (ACE) inhibitors showed elevated D-dimer blood levels, according to a pair of researchers in China. Stopping the medication quickly eased her angioedema symptoms. Her D-dimer levels dropped to normal, too, leading researchers…
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,…
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…
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