News

Orladeyo (berotralstat) — an oral therapy developed by BioCryst Pharmaceuticals to prevent swelling attacks in people with hereditary angioedema (HAE) — is now available to patients, 12 and older, in France, following a decision by a government agency. The French National Agency for Medicines and Health Products Safety granted…

Takeda has submitted an application in Japan seeking regulatory approval of Takhzyro (lanadelumab) for the treatment of hereditary angioedema (HAE). The company’s new drug application to the Japanese Ministry of Health, Labour and Welfare (MHLW) specifically asks Takhzyro be approved for the prophylactic, or preventive, treatment of…

The first patient has been dosed in RAPIDe-1, a Phase 2 trial of PHVS416, an oral on-demand treatment being developed by Pharvaris for swelling attacks in people with hereditary angioedema (HAE). RAPIDe-1 (NCT04618211) will evaluate the investigational therapy’s safety, efficacy, and pharmacokinetics, or how the compound moves…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

BioCryst Pharmaceuticals is seeking the approval of Orladeyo (berotralstat) in the United Kingdom as a preventive treatment for sudden and recurrent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. If granted, Orladeyo would become the first oral therapy — found in trials to be…

Long-term treatment with the approved therapy Haegarda leads to improved quality of life in people with type 1 or 2 hereditary angioedema (HAE), new clinical trial data show. The findings were published in the Orphanet Journal of Rare Diseases, in a study titled “Long-term health-related…

BioCryst Pharmaceuticals’ Orladeyo (berotralstat) has been recommended for approval in the European Union as the first oral therapy for preventing swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. The recommendation was made by the Committee for Medicinal Products for Human Use (CHMP), an arm…

A single dose of PHA121 (PHA-022121), Pharvaris’ investigational therapy for hereditary angioedema (HAE), is safe and can rapidly and sustainably block bradykinin-induced changes in blood parameters, according to two studies in healthy volunteers. According to the company, these findings indicate PHA121 may be well…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…