The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to Intellia Therapeutics’ NTLA-2002, an experimental gene-editing therapy to prevent swelling attacks in people with hereditary angioedema (HAE). A PRIME designation supports the development of experimental therapies that address unmet medical needs. Eligibility for this status is…
A 48-year-old man experienced complete remission from hereditary angioedema (HAE) after undergoing a bone marrow transplant for blood cancer, a case study reports. The transplanted stem cells might have given rise to new cells that acted as a healthy source of C1-inhibitor (C1-INH), the protein that is missing or…
A genetic analysis revealed hereditary angioedema (HAE) was hidden among people with chronic, recurring swelling, but without an apparent family history and normal C1-inhibitor (C1-INH) levels, a study reported. Searching for mutations in patients with chronic, recurring angioedema with an inherited component may detect asymptomatic family members at risk…
Enrollment has begun in APeX-P, a Phase 3 trial designed to evaluate the effects of Orladeyo (berotralstat) in up to 20 children, ages 2–11, with hereditary angioedema (HAE). The open-label study, sponsored by BioCryst Pharmaceuticals, will provide data on Orladeyo’s safety, pharmacological properties, and ability to prevent…
Adult and adolescent patients, ages 12 and older, with hereditary angioedema (HAE) in Israel can now receive once-daily Orladeyo (berotralstat) to prevent HAE-related swelling attacks. The decision to approve the medication by the Israeli Ministry of Health follows similar decisions in Canada and Switzerland, as well as…
A Japanese woman was diagnosed with a rare case of non-episodic angioedema associated with eosinophilia (NEAE), with remarkably high levels of immune eosinophil cells in her bloodstream, a recent case report describes. Swelling in her legs and feet, and associated weight gain, were successfully treated with the anti-inflammatory…
Researchers have developed a simple scoring system to help clinicians identify angioedema patients at high or low risk for intubation, a medical procedure in which a breathing tube is placed into the windpipe through the mouth or nose. Specifically, the need for a breathing tube, called endotracheal…
The Phase 2 KOMPLETE clinical trial testing the ability of oral KVD824 to prevent attacks in people with hereditary angioedema (HAE) has been terminated. The decision by the therapy’s developer, KalVista Pharmaceuticals, was based on the observation of high levels of liver enzymes, a sign of inflammation…
KalVista Pharmaceuticals has launched a two-year open-label extension (OLE) study, dubbed KONFIDENT-S, to test the long-term safety of oral sebetralstat (formerly known as KVD900) as an on-demand treatment for swelling attacks caused by hereditary angioedema (HAE). The study follows the ongoing Phase 3 KONFIDENT clinical trial, involving adults and…
Regulatory authorities in Saudi Arabia have approved daily Orladeyo (berotralstat) to prevent hereditary angioedema (HAE) attacks in adult and adolescent patients, ages 12 and older. “There is a significant need for new treatment options for HAE in Saudi Arabia,” Charlie Gayer, chief commercial officer of BioCryst Pharmaceuticals,…
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