News

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to NTLA-2002, an experimental gene-editing therapy that Intellia Therapeutics is developing to treat hereditary angioedema (HAE). RMAT designation is granted to therapies with the potential to treat, reverse, or cure serious or life-threatening…

Diagnosing and treating hereditary angioedema (HAE) can be challenging, but it is particularly difficult when patients live in rural areas of the U.S., a study based on a survey of allergy and immunology specialists reported. “Misdiagnosis of symptoms, access to a specialist, and affording medications remain…

Garadacimab, a preventive treatment candidate from CSL Behring, led to significant and clinically meaningful reductions in swelling attacks in patients with hereditary angioedema (HAE), according to published data from the Phase 3 VANGUARD clinical trial. The company expects to seek garadacimab’s approval from global health authorities later this…

The Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended Orladeyo (berotralstat) be reimbursed by public drug plans when used for the routine prevention of hereditary angioedema (HAE) attacks in patients 12 years and older. “We are still in need of additional options to help people…

A 61-year-old woman in the U.S. was diagnosed with acquired angioedema and an underlying lymphoproliferative disorder after she sought treatment in an emergency department for sudden tongue swelling and difficulties in talking and swallowing, according to a case study. Lymphoproliferative disorders are characterized by the uncontrolled production of lymphocytes,…

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…