News

Poorly controlled HAE carries heavy burden, affecting life quality

A substantial proportion of adults with hereditary angioedema (HAE) in the Netherlands have poorly controlled disease, significantly affecting their quality of life, a survey study reported. Findings also linked well-controlled HAE to lower medical costs and greater productivity, relative to patients without adequate disease control. “Additional [preventive treatment] strategies…

HAE gene-editing therapy NTLA-2002 receives RMAT designation

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 HAE most challenging across rural US

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…

CSL Behring to seek approval of garadacimab this year

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…

Canadian agency recommends cost of Orladeyo be covered

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…

Elder woman found to have acquired angiodema after tongue swelling

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,…

Orladeyo zeroes swelling attacks in HAE patients

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…