A new generic form of icatibant injection is now available in the United States to treat acute attacks of hereditary angioedema (HAE) in adults age 18 and older. The medicine was licensed to Sandoz by Slayback Pharma for commercial use in the U.S., and comes packaged in…
PHA121, an investigational oral therapy for hereditary angioedema attacks, was well tolerated across multiple doses in a group of healthy volunteers in a Phase 1 trial. Study data also indicates that PHA121 reached therapeutic levels in the first day of a 10-day treatment period, and stable…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
Japan’s Ministry of Health, Labor and Welfare (MHLW) has approved oral Orladeyo (berotralstat), at a daily dose of 150 mg, to prevent hereditary angioedema (HAE) attacks in individuals ages 12 and up. According to the therapy’s developer, BioCryst Pharmaceuticals, Orladeyo is the first prophylactic, or preventive, HAE medication approved…
Variations in the genes KNG1 and XPNPEP1 may be associated with hereditary angioedema (HAE) that occurs in people without any of the mutations that typically cause the disease, a new study suggests. The preliminary findings highlight the genetic complexity of HAE, researchers said. The study, “Deciphering…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a well-deserved…
Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about rare disease communities and salute the creativity of its members. Awardees will be able to display their art and…
Treatment with Takhzyro (lanadelumab) significantly improves health-related quality of life in people with hereditary angioedema (HAE), new data from its pivotal clinical trial show. While all dose regimens improved patient outcomes, the approved dose — 300 mg given every two weeks — provided the greatest benefits. Patients on this…
A case report describes a man with confirmed COVID-19 who developed acute angioedema — a skin symptom that had not previously been associated with the virus — in his lips. “To our knowledge, there has not been…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for a…
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