News

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The U.S. Food and Drug Administration (FDA) has placed a hold on KalVista Pharmaceuticals’ proposed Phase 2 trial of KVD824 — which the developer had expected to launch by the end of June.  The trial is intended to test…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Japan’s National Health Insurance System (NHI) has approved the addition of Orladeyo (berotralstat), an oral preventive treatment for hereditary angioedema (HAE) attacks, to its drug price list. This means that the NHI will reimburse hospitals and pharmacies prescribing Orladeyo to a certain amount under national health insurance programs.

Newer preventive therapies for hereditary angioedema (HAE) lead to a better quality of life and fewer attacks than on-demand treatments, according to a large patient survey study. The study, “Assessing the cost and quality-of-life impact of on-demand‐only medications for adults with hereditary angioedema,” carried out by…

Takhzyro (lanadelumab) is more effective than Cinryze at preventing swelling attacks in people with hereditary angioedema (HAE), a new study indicates. Analyses suggested that treatment with Takhzyro reduced the rates of HAE attack by 46–73% as compared with Cinryze in an indirect comparison of previous studies. Titled…

The experimental therapy IONIS-PKK-LRx can significantly lower the number of monthly swelling attacks in patients with hereditary angioedema (HAE) type 1 and 2, according to top-line data from an ongoing Phase 2 trial. Newly announced data show the study (NCT04030598), sponsored by therapy developer Ionis Pharmaceuticals,…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Haegarda, CSL Behring’s preventive treatment for hereditary angioedema (HAE) attacks, will be available to eligible HAE patients across Canada starting on April 6. The availability announcement was issued by Canadian Blood Services (CBS), the country’s blood authority, which is responsible for managing the blood supply in all…

Off-label C1 inhibitor concentrate successfully treated people with severe acute angioedema triggered by the use of so-called ACE inhibitor medications, a small study has found. The study, “Effectiveness of C1-INH therapy in angiotensin converting…