The European Commission has granted orphan medicinal product designation (OMPD) to navenibart, an experimental long-acting therapy for hereditary angioedema (HAE). The designation is aimed at providing incentives for the development of therapies that are meant to diagnose, prevent, or treat rare diseases, or those affecting fewer than 10,000 people…
Preventing swelling attacks in children in India with hereditary angioedema (HAE) relies mostly on using tranexamic acid (TA) and attenuated androgens, or male hormones, due to the lack of accessible first-line treatment options, a single-center study shows. Neither therapy is preferred for HAE and attenuated androgens are contraindicated…
Intellia Therapeutics has launched a Phase 3 clinical trial to evaluate the efficacy and safety of NTLA-2002, a single-dose gene-editing therapy for people with hereditary angioedema (HAE). The new trial, dubbed HAELO (NCT06634420), was given the green light by the U.S. Food and Drug Administration (FDA) based…
Kalvista Pharmaceuticals has submitted applications to regulatory authorities in the U.K., Switzerland, Australia, and Singapore requesting the approval of sebetralstat, an oral, on-demand treatment for hereditary angioedema (HAE) in patients ages 12 and older. The four applications were submitted through the Access Consortium framework, a coalition of…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to navenibart — the new generic name of Astria Therapeutics’ experimental therapy STAR-0215 — as a treatment for hereditary angioedema (HAE).
People with hereditary angioedema (HAE) in Japan with access to on-demand therapies alone can have a high rate of attack frequency — weekly or monthly — that associates with a poorer quality of life, a study in that country reports. Patients’ functioning, fears, and feelings of shame significantly linked…
A man who experienced recurrent episodes of tongue swelling was diagnosed with angioedema related to the use of an angiotensin-converting enzyme (ACE) inhibitor to treat high blood pressure, according to a case report. The patient’s symptoms eased after stopping treatment with the medication, lisinopril, with no recurrence of angioedema…
Treatment with Takhzyro (lanadelumab), an injectable antibody therapy, led to a mean reduction of 94.7% in monthly hereditary angioedema (HAE) attacks in adolescents with HAE, according to findings of a Phase 3 extension study. These results, which focused on a subgroup of 21 patients with type 1…
Orladeyo (berotralstat), an approved oral treatment to prevent attacks in hereditary angioedema (HAE) patients 12 years and older, is one step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Biocryst Pharmaceuticals. Biocryst has successfully completed negotiations with the pan-Canadian…
The case of an elderly man who developed acquired angioedema associated with an underlying blood disorder called monoclonal gammopathy was described in a new report, with the researchers noting the condition is “very rare but serious.” The man’s angioedema was controlled only after the underlying monoclonal gammopathy was successfully…
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