The National Institute for Health and Clinical Excellence (NICE), the health technology appraisal body in England and Wales, has issued its Final Appraisal Determination (FAD) recommending Takhzyro (lanadelumab) subcutaneous injection as an option for preventing recurrent attacks of HAE in patients aged 12 years and older only if: (1) they are eligible for preventive C1-esterase [...]
The clinical-stage company Pharvaris B.V. which is focused on the discovery and development of novel oral B2-receptor antagonists for the treatment of HAE, has completed a 66 million USD Series B financing. In addition, the company announces its entry into clinical development with the initiation of a Phase 1 study of its lead compound, PHA121, in [...]
At the presentation of the financial results for the quarter ended 30 June 2019, Kenneth T. Mills, President and CEO of the clinical-stage biotechnology company REGENXBIO Inc., said: "We recently announced the expansion of our pipeline using NAV Vectors to deliver therapeutic antibodies, beginning with novel treatments for HAE. This approach has enormous potential for patients [...]
BioCryst Pharmaceuticals, Inc. has announced the financial results for the second quarter ended 30 June 2019 and provided a corporate update. “We have just returned from the 2019 HAEA National Patient Summit with more than 1,000 attendees from the U.S. and around the world. Patients’ excitement about BCX7353 was resounding as they told us an oral [...]
A ‘one-of-a-kind’ medication access program from HAE International is now successfully delivering potentially life-saving medication to HAE-patients in South Africa. “As no modern medications have been commercially available in South Africa until now, HAE patients have been unable to access them. The HAE International Global Access Program offers a regulatory compliant process that allows [...]
Dear HAE friends, Welcome to the second 2019 issue of the HAEi magazine Global Perspectives. This time you will, among many other items, find information on: News from the HAEi Regional Patient Advocates Life-saving medication to South African HAE patients Another success on the road to Santiago 11th C1-INH Deficiency & Angioedema Workshop including summaries [...]
HAE International extends a very warm thank you to each and everyone who took part in the HAE Global Walk from 1 April to 31 May 2019 – once more proving that this is a truly worldwide HAE event. “First launched in 2016 the HAE Global Walk registered more than 12,000,000 steps. In 2017 the [...]
Takeda Pharmaceutical Company Limited presents new data from an ad-hoc analysis of the Phase 3 HELP Study™, designed to evaluate the onset of action for TAKHZYRO (lanadelumab) during days 0-69 of treatment. The analysis suggests that TAKHZYRO starts to prevent HAE attacks during this early treatment phase, with patients experiencing an 80.1% decrease in mean [...]
At the C1 Inhibitor Deficiency and Angioedema Workshop in Budapest, Hungary earlier this month KalVista Pharmaceuticals, Inc. presented new data regarding its KVD900 project. “KVD900 is an oral, novel, potent and selective inhibitor of plasma kallikrein, a validated target in HAE,” said Andrew Crockett, CEO of KalVista. “We believe KVD900 represents a new therapeutic opportunity [...]
The randomized (n=121), double-blind, placebo-controlled, Phase 3 APeX-2 trial of once-daily, oral BCX7353 for the prevention of HAE attacks has achieved its primary endpoint for both dose levels (110 mg and 150 mg), with the 150 mg dose reducing the attack rate in HAE patients by 44 percent (p<0.001) compared to placebo. Fifty percent of [...]