Protease-activated receptor 1 (PAR1), a protein involved in the regulation of blood vessel permeability or leakage, may contribute to hereditary angioedema (HAE) attacks. That is the preliminary conclusion of a study reporting evidence suggesting that PAR1 was highly activated in the throat tissue of a woman with HAE type…
The U.S. Food and Drug Administration (FDA) has cleared Astria Therapeutics to move STAR-0215, its experimental treatment for preventing swelling attacks in hereditary angioedema (HAE), into clinical testing. With the positive decision on its investigational new drug (IND) application, the company is now planning to launch a Phase…
CSL Behring has won an award for its efforts to ensure patient access to therapies for hereditary angioedema (HAE). The company received the nod in the Biologics & Injectables category of the Patient Access Awards, launched this year by Managed Markets Insight & Technology (MMIT), a provider of…
One person out of six treated with Ruconest (rhC1INH) for idiopathic non-histaminergic angioedema (InH-AAE) as part of an exploratory study showed a positive clinical response to the medication. The findings may mean that Ruconest is effective in a subset of InH-AAE patients, the researchers noted. The study, “…
Dermatologists, pediatricians, and emergency doctors may be more aware of hereditary angioedema (HAE) than doctors of other specialties, according to an online survey conducted in Japan. This discrepancy among healthcare professionals highlights the importance of educational efforts and specialist accreditation in raising awareness about the condition within the medical…
New mothers may safely take Firazyr (icatibant; generics available) to treat swelling attacks due to hereditary angioedema (HAE) and continue to breastfeed, provided they wait at least six hours after the last dose before giving their baby breast milk, a case report from Japan suggested. Icatibant’s safety in women…
STAR-0215, an investigational therapy to prevent swelling attacks in people with hereditary angioedema (HAE), rapidly and durably suppressed the enzyme kallikrein, supporting a once every three months or longer dosing in humans, according to new preclinical data. The data was recently presented in a flash talk titled, “…
Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
Treatment with sebetralstat, an investigational and oral on-demand therapy, resulted in rapid relief for both abdominal and peripheral attacks in adults with hereditary angioedema (HAE), according to a new analysis Phase 2 trial data. Sebetralstat (formerly KVD900), by KalVista Pharmaceuticals, is a small molecule inhibitor of the…
Switching from under-the-skin injections of Takhzyro (lanadelumab) to oral capsules of Orladeyo (berotralstat) was generally safe and kept the occurrence of swelling attacks at low rates over one year in people with hereditary angioedema (HAE), according to a new data analysis of the Phase 2/3 APeX-S trial.
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