The U.S. Food and Drug Administration (FDA) has given Intellia Therapeutics permission to open U.S. sites in its ongoing clinical trial of NTLA-2002, an investigational gene-editing therapy for hereditary angioedema (HAE). Specifics are not yet available on the number or location of U.S. sites for the global Phase…
People with hereditary angioedema (HAE) who took Orladeyo (berotralstat) for up to two years experienced no swelling attacks for most of the treatment duration, with fewer swelling attacks translating into a better quality of life. That’s according to results from APeX-S and APeX-2, two clinical studies that evaluated…
A genetic analysis revealed hereditary angioedema (HAE) was hidden among people with chronic, recurring swelling, but without an apparent family history and normal C1-inhibitor (C1-INH) levels, a study reported. Searching for mutations in patients with chronic, recurring angioedema with an inherited component may detect asymptomatic family members at risk…
Treatment with Takhzyro (lanadelumab) led to a more than 80% reduction in swelling attacks in a woman with hereditary angioedema (HAE) and normal C1-inhibitor (C1-INH) activity, a case study reports. The medication effectively reduced both the frequency and severity of the attacks, leading to ongoing benefits for this…
The number of cases of hereditary angioedema with normal C1 inhibitor (HAE-nl-C1INH) within the U.S. was estimated to be between 1,230 and 1,331 from May 2019 and April 2020, according to a survey conducted among U.S. physicians. Patients waited a mean of six years to receive a diagnosis, which…
A 70-year-old woman diagnosed with non-episodic angioedema associated with eosinophilia (NEAE) — high levels of immune eosinophil cells — was successfully treated with reslizumab, a medication normally used to treat asthma associated with eosinophilia, according to a case report. Her symptoms had started after receiving a…
The U.S. Food and Drug Administration (FDA) has extended the approval of Takhzyro (lanadelumab) to include children with hereditary angioedema (HAE) starting at age 2. Takhzyro, available to patients ages 12 and older in the U.S. since 2018, is now the only approved treatment to prevent HAE swelling…
Astria Therapeutics has initiated a Phase 1b/2 trial called ALPHA-STAR to test STAR-0215, the company’s investigational therapy to prevent swelling attacks in people with hereditary angioedema (HAE). ALPHA-STAR (NCT05695248) intends to enroll 18 adults with HAE types 1 or 2 who have had at least four swelling…
Despite long-term preventive treatment, a 38-year-old woman with hereditary angioedema (HAE) with normal C1-inhibitor levels had severe episodes of disease worsening, as described in a recent case report. The life-threatening sequelae and decreased quality of life illustrate the need for more research with this rare form of HAE, according…
BioCryst Pharmaceuticals has tapped Swixx BioPharma to commercialize the hereditary angioedema (HAE) treatment Orladeyo (berotralstat) in central and Eastern Europe. Agreement terms call for Swixx, a Switzerland-based biopharmaceutical company, to handle commercialization of the oral therapy in 15 markets. “We continue to build partnerships with companies…
Get regular updates to your inbox.