NTLA-2002, an experimental gene-editing therapy for hereditary angioedema (HAE) being developed by Intellia Therapeutics, has been awarded an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), the company announced. This designation is the first step in the U.K.’s Innovative Licensing and Access…
Most hereditary angioedema (HAE) patients in a Phase 1/2 clinical trial have gone months without a swelling attack following a single infusion of the experimental gene-editing therapy NTLA-2002. The remaining patients haven’t yet reached the pre-specified 16-week follow-up period for their attack rates to be analyzed, but their comparable…
People with hereditary angioedema (HAE) who switched to Orladeyo (berotralstat) from other preventive treatments generally experience a reduction in swelling attacks. That is according to real-world data presented by BioCryst Pharmaceuticals, Orladeyo’s developer, during the 2022 Annual Scientific Meeting of the American College of Allergy, Asthma…
The U.S. Food and Drug Administration (FDA) has agreed to review an application from Takeda to extend the approval of Takhzyro (lanadelumab) for children as young as 2 with hereditary angioedema (HAE). The application has been given priority review status and a decision from is expected in…
Japan’s Ministry of Health, Labour and Welfare has approved Berinert S.C. Injection 2000 — a C1-inhibitor replacement therapy designed to be self-administered at home via injection under the skin twice per week — for preventing swelling attacks in people with hereditary angioedema (HAE). The therapy has been approved in…
The European Patent Office has granted patent protection to Verseon covering the company’s experimental plasma kallikrein inhibitors (PKIs). Two of these investigational medications, called VE-4666 and VE-4062, are currently in preclinical development as potential treatments of hereditary angioedema (HAE). “We’re very pleased with the European Patent Office’s…
NTLA-2002, an experimental gene-editing therapy designed to prevent swelling attacks in people with hereditary angioedema (HAE), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). This designation is given to investigational treatments that have the potential to improve care for rare diseases, defined as…
Protease-activated receptor 1 (PAR1), a protein involved in the regulation of blood vessel permeability or leakage, may contribute to hereditary angioedema (HAE) attacks. That is the preliminary conclusion of a study reporting evidence suggesting that PAR1 was highly activated in the throat tissue of a woman with HAE type…
Most young children with hereditary angioedema (HAE) in the open-label Phase 3 SPRING study were free of swelling attacks following a year of treatment with Takhzyro (lanadelumab). That’s according to new data recently shared at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2022 in…
Many people with hereditary angioedema (HAE) have non-swelling symptoms, such as rash or fatigue, even when they aren’t having an acute swelling attack, a new study highlights. “HAE symptoms are not limited to only those experienced during HAE swelling attacks. Many different symptoms (including abdominal symptoms, pain, headache, fatigue, depression, and…
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