News

Genetic analysis reveals recurrent HAE with normal C1-INH: Study

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…

Patient with HAE and normal C1-INH treated with Takhzyro

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…

Survey of US doctors estimates prevalence of HAE-nl-C1INH

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…

Phase 1b/2 trial of STAR-0215 is now enrolling HAE patients

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…

Woman with HAE, normal C1-INH has attacks despite preventive care

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, Swixx BioPharma Partner on Orladeyo in Europe

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…

APeX-P Trial of Orladeyo Now Enrolling Children 2–11 With HAE

Enrollment has begun in APeX-P, a Phase 3 trial designed to evaluate the effects of Orladeyo (berotralstat) in up to 20 children, ages 2–11, with hereditary angioedema (HAE). The open-label study, sponsored by BioCryst Pharmaceuticals, will provide data on Orladeyo’s safety, pharmacological properties, and ability to prevent…

HAE Therapy ADX-324 Enters Phase 1 Clinical Testing

The first group of participants has been dosed in a Phase 1 clinical trial evaluating the safety and pharmacological properties of ADX-324, an experimental treatment for hereditary angioedema (HAE) that is being developed by Adarx Pharmaceuticals. “Dosing of our first participants in this trial is a major milestone…