BioCryst Pharmaceuticals has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) requesting the approval of berotralstat (BCX7353), a once daily, oral treatment to prevent swelling attacks in people with hereditary angioedema (HAE). The latest results of APeX-S and APeX-2…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Factors involved in blood vessel permeability — called VEGF-A, VEGF-C, and sPLA2 — may predispose people who take ACE inhibitor medications for high blood pressure to develop non-allergic angioedema, a study reports. Moreover, the findings indicate that switching to sartans, another class of antihypertensive medicines,…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
The Canadian Drug Expert Committee (CDEC) has recommended that Takhzyro (lanadelumb) be reimbursed by public drug plans when used as a preventive treatment for hereditary angioedema (HAE) attacks in patients 12 and older. “HAE is a chronic disease that results in acute attacks of swelling…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
BioCryst Pharmaceuticals has licensed rights to Torii Pharmaceutical for the commercialization of BCX7353 — an oral, once-daily investigational therapy for the prevention of hereditary angioedema (HAE) attacks — in Japan, pending the treatment’s approval. The plan is for BioCryst to submit a Japanese new drug application (JNDA)…
Having systemic lupus erythematosus (SLE) can considerably increase the likelihood of developing angioedema, a new study suggests. Women, African-Americans, and younger patients may be particularly susceptible, its findings show. The research, “Systemic Lupus Erythematosus and Angioedema: A Cross-Sectional Study From the National Inpatient Sample,” was published in the…
Acquired angioedema can be the first symptom of underlying conditions such as lymphoma. In those cases, cancer treatment may be the best approach to stop angioedema manifestations, a case report suggests. The study, “A Case of Acquired Angioedema with Low C1 Inhibitor (C1-INH) Associated with…
Kalbitor (ecallantide) is a potential treatment for angioedema attacks that do not respond to medication, in adolescents who also have systemic lupus erythematosus (SLE), even if they have normal levels of C1 inhibitor protein, two case reports suggest. The study combining the reports, “Ecallantide:…
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