News

Diagnosis of HAE delayed a median of 20 years in Latvia: Study

People with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) typically experience a significantly delayed diagnosis — even with a family history positive for the disease, according to a nationwide study in Latvia. Indeed, the median delay in obtaining a diagnosis in the Northern European country is 20.5 years, researchers…

PHVS416 shows promise in treating HAE swelling attacks: Trial data

Pharvaris’ PHVS416, a soft-gel capsule of PHA121, now also known as deucrictibant, continues to show promising safety and effectiveness for treating swelling attacks in people with hereditary angioedema (HAE). That’s according to data from RAPIDe-1 (NCT04618211), a Phase 2 clinical trial that’s comparing on-demand treatment with PHVS416…

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…